Abstract / Synopsis:

AAV8-RPGR gene therapy for X-linked retinitis pigmentosa showed early responses to treatment at one month with increased retinal sensitivity with retinal toxicity.

This article was reviewed by Paulo E. Stanga, MD

The results of treatment of X-linked retinitis pigmentosa (XLRP) with AAV8-RPGR gene therapy proved to be early and effective with durable improvements in vision occurring as early as one month following treatment.

XLRP, a rare disease that comprises 10% to 20% of RP worldwide, affects mostly males, and 70% of the cases are caused by RPGR gene mutations and 60% of those are caused by gene mutations in RPGR-ORF15. In this form of RP, the median age of blindness is 45 years, which is younger than the other forms.

Disease progression occurs in stages, with nyctalopia manifesting in the early stage, peripheral visual field constriction in the middle stage, and central visual deterioration and visual loss in the end stage, according to Paulo E. Stanga, MD, professor of Ophthalmology and Retinal Regeneration, Manchester Royal Eye Hospital and University of Manchester, London, UK.

Related: Gene therapy offering hope for retinal, corneal patients

The RPGR mutations cause abnormal transport across the cilium, where RPGR is located, and this abnormal transport results in photoreceptor death.

Obviously, this leads to loss of retinal sensitivity across the visual field and loss of visual acuity, he said.

The treatment that Dr. Stanga and colleagues devised has the goal of correcting the full length of the RPGR-ORF15 mRNA.

We aim for yields of high expression levels that are four times higher than the expression levels of the wild-type RPGR, he explained.

Related: Research targets precision data for gene, cell therapy

Six-month Phase I resultsDr. Stanga and colleagues are currently conducting a two-year dose-escalation clinical trial. The study included men who were 18 years and older with genetically confirmed XLRP. All patients had active disease that was visible bilaterally in the maculas. The study included six cohorts, with the following levels of affect vision: 1, better than light perception; 2 and 3, 34 to 73 Early Treatment Diabetic Retinopathy Study (ETDRS) letters; and 4, 5, and 6, greater than 34 ETDRS letters.

The primary endpoint was the incidence of dose-limiting toxicities and treatment-emergent adverse events. The secondary endpoints were the changes in microperimetry, visual stability, and changes in the ellipsoidal zone on spectral-domain optical coherence tomography, Dr. Stanga recounted.

The patients underwent a surgical procedure that included creation of a bleb followed by injection of the virus vector within the bleb.

Related: New vitreoretinal tools advancing surgical outcomes

The investigators evaluated the early effects of changes in the retinal sensitivity in the central retina using microperimetry (Maia, Centervue). The central 16 retinal loci represent 8 degrees of vision; an improvement of five of the central 16 loci equals a 30% improvement in the central visual field. An improvement of 7 dB represents five times greater light sensitivity, he explained.

One month after treatment, Dr. Stanga reported that there was a significant improvement in microperimetry in six of the 12 treated eyes in cohorts 3 to 6 that occurred at one month after vector injection; these cohorts received therapeutic doses. Cohorts 1 and 2, which received subtherapeutic doses, showed no changes.

Cohort 3 showed a mean improvement in the mean retinal sensitivity of 5 to 6 dB in the central 16 retinal loci between the treated and untreated eyes. The improvement became apparent at one month and remained relatively stable at three and six months, Dr. Stanga reported.

According to Dr. Stanga, these changes in retinal sensitivity differed from those observed in untreated eyes in the central 16 retinal loci. The untreated eyes showed decreases in retinal sensitivity over time.

Related: Surgeons provides pearls for handling retinal tears

The microperimetry heat maps also reflected the changes in the treated eyes with enlargement of the sensitive areas.

The investigators also reported that they also determined that the gene therapy with AAV8-RPGR gene therapy for XLRP was generally well tolerated.

No patients left the study and no dose-limiting toxicities were readily apparent.

Transient inflammation developed in the higher cohorts that responded to systemic steroid therapy. Two ocular adverse effects were related to the procedure or drug.

ConclusionsWe demonstrated proof of concept with durable dose-related improvements that appeared as early as one month after treatment across multiple microperimetry analyses, Dr. Stanga concluded. The preliminary efficacy signals were exhibited in cohorts 3 to 6, which responded to the highest doses.

Read more by Lynda Charters

Paulo E. Stanga, MDE: [emailprotected]This article is based on Dr. Stanga's presentation at the American Academy of Ophthalmology 2019 annual meeting. Dr. Stanga has no financial interest in this subject matter.

Excerpt from:
Gene therapy offers treatment for X-linked retinitis pigmentosa - Modern Retina

Recommendation and review posted by Bethany Smith

AUSTIN, Texas & CAMBRIDGE, Mass. (Feb. 11, 2020) Genprex, Inc. ("Genprex" or the "Company") (NASDAQ: GNPX), a clinical-stage gene therapy company developing potentially life-changing technologies for patients with cancer and other serious diseases, today announced that it signed an exclusive license agreement with the University of Pittsburgh for a diabetes gene therapy that may have the potential to cure Type 1 and Type 2 diabetes, which together currently affect approximately 30.3 million people in the U.S, or 9 percent of the U.S. population.

The diabetes gene therapy, which was developed by lead researcher and Harvard graduate, Dr. George Gittes, at the Rangos Research Center at UPMC Children's Hospital of Pittsburgh, works by reprogramming beta cells in the pancreas to restore their function, thereby replenishing levels of insulin. The novel infusion process uses an endoscope and an adeno-associated virus (AAV) vector to deliver Pdx1 and MafA genes to the pancreas. The proteins these genes express transform alpha cells in the pancreas into functional beta-like cells, which can produce insulin but are distinct enough from beta cells to evade the body's immune system.

The diabetes gene therapy has been tested in vivo in mice and nonhuman primates. In studies of diabetic mice, the gene therapy approach restored normal blood glucose levels for an extended period of time, typically around four months. According to Dr. Gittes, the duration of restored blood glucose levels in mice could translate to decades in humans. Following preclinical studies, Dr. Gittes and his team plan to begin a Phase I clinical trial in diabetic patients, which could be the first-ever gene therapy tested in humans for diabetes.

"One of the biggest advantages of this gene therapy is that it could eliminate the need for insulin replacement therapy for diabetic patients," said Dr. Gittes. "Lifting this huge burden for the millions of patients who must continuously monitor blood glucose levels and inject insulin daily would be a breakthrough in modern medicine. This therapy has the potential to truly disrupt the diabetes market."

Genprex will add this exciting technology to its research and development pipeline, diversifying its portfolio and expanding its clinical development programs. The company will continue its focus on developing its immunogene therapies for cancer, including Oncoprex immunogene therapy, its lead drug candidate for non-small cell lung cancer, in parallel with development of the new diabetes gene therapy.

"We are excited to announce the licensing agreement with The University of Pittsburgh, and we look forward to working with Dr. Gittes and his team to develop this groundbreaking treatment for diabetes," said Rodney Varner, Genprex's Chairman and Chief Executive Officer. "At Genprex, we have always put patient needs first, focusing on ways to bring new treatment options to patient populations who have large unmet medical needs. We believe this diabetes gene therapy may potentially become a new treatment option for the millions of diabetes patients who now must take insulin replacement therapy, and it may be effective for patients who do not benefit sufficiently from that therapy. Even more moving, the diabetes gene therapy could hold the potential to provide long term effectiveness, or even be a cure, for diabetes patients."

Genprex plans to pursue potential partnerships for the development of this therapy globally and in the U.S.

According to the American Diabetes Association, more than 30 million Americans have diabetes, and approximately 1.5 million Americans are diagnosed with diabetes every year. Diabetes patients have the continuous burden of checking and monitoring their blood glucose levels and injecting insulin on a daily basis. Without effective management of diabetes, patients are at risk of stroke, hyperglycemia, cardiovascular disease, diabetic ketoacidosis and extremity amputation. Diabetes is the seventh leading cause of death in the U.S.

About Genprex, Inc.

Genprex, Inc. is a clinical-stage gene therapy company developing potentially life-changing technologies for patients with cancer and other serious diseases. Genprex's technologies are designed to administer disease-fighting genes to provide new treatment options for large patient populations with cancer and other serious diseases who currently have limited treatment options. Genprex works with world-class institutions and collaborators to in-license and develop drug candidates to further its pipeline of gene therapies in order to provide novel treatment approaches for patients with cancer and other serious diseases. The company's lead product candidate, Oncoprex immunogene therapy for non-small cell lung cancer (NSCLC), uses the company's unique, proprietary platform which delivers cancer-fighting genes by encapsulating them into nanoscale hollow spheres called nanovesicles, which are then administered intravenously and taken up by tumor cells where they express proteins that are missing or found in low quantities. In January 2020, the FDA granted Fast Track Designation for Oncoprex in combination with AstraZeneca's Tagrisso for the treatment of NSCLC. For more information, please visit the company's website at http://www.genprex.com or follow Genprex on Twitter, Facebook and LinkedIn.

Forward-Looking Statements

Statements contained in this press release regarding matters that are not historical facts are "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Such statements include, but are not limited to, statements regarding the effects of the licensed gene therapy on diabetes and the effect of Genprex's other product candidates, alone and in combination with other therapies, on cancer, as well as Genprex's ongoing and planned preclinical and clinical studies and potential partnerships. Risks that contribute to the uncertain nature of the forward-looking statements include risks relating to the effects of the safety and effectiveness of the licensed gene therapy and of Genprex's other product candidates, alone and in combination with other therapies, as well as the success of Genprex's ongoing and planned preclinical and clinical studies and the success of Genprex's efforts in concluding potential partnering arrangements for product development and commercialization. Other risks and uncertainties associated with Genprex and its product candidates are described more fully under the caption "Risk Factors" and elsewhere in Genprex's filings and reports with the United States Securities and Exchange Commission. All forward-looking statements contained in this press release speak only as of the date on which they were made. Genprex undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made.

Read more here:
Genprex and University of Pittsburgh Sign License Agreement - Patch.com

Recommendation and review posted by Bethany Smith

Neurocognitive development of young MPS IIIA patients preserved up to two years post ABO-102 treatment

Dose-dependent and sustained reductions in disease-specific biomarkers denotes clear biologic effects of ABO-102 and ABO-101

First patient treated in cohort 3 of ABO-101 MPS IIIB trial; total enrollment eight patients

Favorable safety profile observed in both studies

NEW YORK and CLEVELAND, Feb. 12, 2020 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (Nasdaq: ABEO), a fully-integrated leader in gene and cell therapy, today announced that researchers from the Abigail Wexner Research Institute (AWRI) at Nationwide Childrens Hospital presented positive interim data from two ongoing Phase I/II clinical trials evaluating ABO-102 and ABO-101, the Companys investigational gene therapies for MPS IIIA and MPS IIIB, respectively, at WORLDSymposium. Results from the Transpher A study demonstrated that MPS IIIA patients younger than 30 months treated with ABO-102 in dose cohort 3 continue to show neurocognitive development 18 months to two years after treatment. Reductions in cerebrospinal fluid (CSF) heparan sulfate (HS), denoting enzyme activity in the central nervous system, and liver volume reductions remain stable two years after treatment. Results from the Transpher B study showed that ABO-101 also improved multiple disease biomarkers providing clear evidence of a biologic effect in patients with MPS IIIB. Dosing in cohort 2 is complete and the first patient in cohort 3 was treated in late January, with a total of 8 patients treated to date. Both therapies have been well-tolerated to date. Abeona licensed the AAV9-based gene therapy technology underpinning ABO-102 and ABO-101 from AWRI at Nationwide Childrens where it was developed.

Todays presentations are available on abeonatherapeutics.com by following this link:https://investors.abeonatherapeutics.com/news-events

In total, the new results continue to show that early treatment with ABO-102 can help preserve neurodevelopment in children with MPS IIIA. These data will inform our ongoing discussions with the FDA and EMA, as we work towards providing a regulatory update in the second quarter, said Joo Siffert, M.D., Chief Executive Officer. For ABO-101, the reductions in disease-specific biomarkers are encouraging and demonstrate a clear biologic effect, which parallels that seen in the MPS IIIA study. We look forward to enrolling the Transpher B study as expeditiously as possible.

Results from the Transpher A study, an ongoing Phase I/II clinical trial with ABO-102 showed that:

The interim results presented today add to evidence suggesting a single intravenous dose of ABO-102 AAV9-based gene therapy has the potential to help MPS IIIA patients sustain neurocognitive development when they are treated at a young age, said Kevin Flanigan, M.D., Director, Center for Gene Therapy at AWRI at Nationwide Children's and Transpher A study investigator. These data showed that ABO-102 can deliver a functional copy of the SGSH gene to cells of the CNS and peripheral organs, as evidenced by the clinical benefits in neurocognition and biophysical measures and improvements in disease-specific biomarkers.

Sites in the U.S., Spain, and Australia continue to enroll eligible patients into the Transpher A study. Additional information about the trial is available at AbeonaTrials.com and ClinicalTrials.gov.

Results from cohorts 1 and 2 (n=7) of the Transpher B study, an ongoing Phase I/II clinical trial showed that ABO-101 treatment demonstrated biologic effect in patients with MPS IIIB, as evidenced by initial improvements in multiple disease biomarkers associated with abnormal accumulation of glycosaminoglycans (GAGs) in the brain and throughout the body:

The Transpher B study provides hope that we may one day alter the course of this devastating disease, said Kim McBride, M.D., Principal Investigator at AWRI at Nationwide Children's and co-investigator for the Transpher B study. The impact on disease biomarkers in the early stages of follow up suggest the potential of ABO-101 gene therapy to break down the accumulation of glycosaminoglycans that underlie MPS IIIB pathology. I look forward to working with fellow investigators to gather more data from the study, including results from high-dose cohort 3.

Dose cohort 2 has been completed and dosing is underway in cohort 3 (n=1). Sites in the U.S., Spain, and France continue to enroll eligible patients into the Transpher B study. Additional information about the trial is available at abeonatherapeutics.com/clinical-trials and ClinicalTrials.gov.

About The Transpher A StudyThe Transpher A Study (NCT02716246) is an ongoing, two-year, open-label, dose-escalation, Phase I/II global clinical trial assessing ABO-102 for the treatment of patients with Sanfilippo syndrome type A (MPS IIIA). The study, also known as ABT-001, is intended for patients 6 months to 2 years of age, or patients older than 2 years with a cognitive Developmental Quotient of 60% or above. The study has enrolled 14 patients to date across three dose-escalating cohorts (N=3, N=3, N=8) and remains open for enrollment. The gene therapy ABO-102 is delivered using AAV9 technology via a single-dose intravenous infusion. The study primary endpoints are neurodevelopment and safety, with secondary endpoints including behavior evaluations, quality of life, enzyme activity in cerebrospinal fluid (CSF) and plasma, heparan sulfate levels in CSF, plasma and urine, and brain and liver volume.

About The Transpher B StudyThe Transpher B Study (NCT03315182) is an ongoing, two-year, open-label, dose-escalation, Phase I/II global clinical trial assessing ABO-101 for the treatment of patients with Sanfilippo syndrome type B (MPS IIIB). The study, also known as ABT-002, is intended for patients 6 months to 2 years of age, or patients older than 2 years with a cognitive Developmental Quotient of 60% or above. The study has enrolled 8 patients to date across three dose-escalating cohorts (N=2, N=5, N=1) and remains open for enrollment. The gene therapy ABO-101 is delivered using AAV9 technology via a single-dose intravenous infusion. The study primary endpoints are neurodevelopment and safety, with secondary endpoints including behavior evaluations, quality of life, enzyme activity in cerebrospinal fluid (CSF) and plasma, heparan sulfate levels in CSF, plasma and urine, and brain and liver volume.

About ABO-102ABO-102 is a novel gene therapy in Phase I/II development for Sanfilippo syndrome type A (MPS IIIA), a rare lysosomal storage disease with no approved treatment that primarily affects the central nervous system (CNS). ABO-102 is dosed in a one-time intravenous infusion using a self-complementary AAV9 vector to deliver a functional copy of the SGSH gene to cells of the CNS and peripheral organs. The therapy is designed to address the underlying SGSH enzyme deficiency responsible for abnormal accumulation of glycosaminoglycans in the brain and throughout the body that results in progressive cell damage and neurodevelopmental and physical decline. In the U.S., Abeona holds Regenerative Medicine Advanced Therapy, Fast Track, Rare Pediatric Disease, and Orphan Drug designations for the ABO-102 clinical program. In the EU, the Company holds PRIME and Orphan medicinal product designations.

About ABO-101ABO-101 is a novel gene therapy in Phase I/II development for Sanfilippo syndrome type B (MPS IIIB), a rare lysosomal storage disease with no approved therapy that primarily affects the central nervous system (CNS). ABO-101 is dosed in a one-time intravenous infusion using a self-complementary AAV9 vector to deliver a functional copy of the NAGLU gene to cells of the CNS and peripheral tissues. The therapy is designed to address the underlying NAGLU enzyme deficiency responsible for abnormal accumulation of glycosaminoglycans in the brain and throughout the body that results in progressive cell damage and neurodevelopmental and physical decline. In the U.S., Abeona holds Fast Track and Rare Pediatric Disease designations for ABO-101 and Orphan Drug designation in both the U.S. and EU.

About Sanfilippo Syndrome Type A (MPS IIIA)Sanfilippo syndrome type A (MPS IIIA) is a rare, fatal lysosomal storage disease with no approved treatment that primarily affects the CNS and is characterized by rapid neurodevelopmental and physical decline. Children with MPS IIIA present with progressive language and cognitive decline and behavioral abnormalities. Other symptoms include sleep problems and frequent ear infections. Additionally, distinctive facial features with thick eyebrows or a unibrow, full lips and excessive body hair for ones age, and liver/spleen enlargement are also present in early childhood. MPS IIIA is caused by genetic mutations that lead to a deficiency in the SGSH enzyme responsible for breaking down glycosaminoglycans, which accumulate in cells throughout the body resulting in rapid health decline associated with the disorder.

About Sanfilippo syndrome type B (MPS IIIB)Sanfilippo syndrome type B (MPS IIIB) is a rare and fatal lysosomal storage disease with no approved therapy that primarily affects the central nervous system and is characterized by rapid neurodevelopmental and physical decline. Children with MPS IIIB present with progressive language and cognitive decline and behavioral abnormalities. Other symptoms include sleep problems and frequent ear infections. Additionally, distinctive signs such as facial features with thick eyebrows or a unibrow, full lips and excessive body hair for ones age and liver/spleen enlargement are also present. The underlying cause of MPS IIIB is a deficiency in the NAGLU enzyme responsible for breaking down glycosaminoglycans, which accumulate throughout the body resulting in rapid decline associated with the disorder.

About Abeona TherapeuticsAbeona Therapeutics Inc. is a clinical-stage biopharmaceutical company developing gene and cell therapies for serious diseases. The Companys clinical programs include EB-101, its autologous, gene-corrected cell therapy for recessive dystrophic epidermolysis bullosa, as well as ABO-102 and ABO-101, novel AAV9-based gene therapies for Sanfilippo syndrome types A and B (MPS IIIA and MPS IIIB), respectively. The Companys portfolio of AAV9-based gene therapies also features ABO-202 and ABO-201 for CLN1 disease and CLN3 disease, respectively. Abeona has received numerous regulatory designations from the FDA and EMA for its pipeline candidates, including Regenerative Medicine Advanced Therapy designation for two candidates (EB-101 and ABO-102). http://www.abeonatherapeutics.com

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Abeona Therapeutics Announces Positive Interim Data from MPS III Gene Therapy Programs Presented at WORLDSymposium - PharmiWeb.com

Recommendation and review posted by Bethany Smith

North Carolina is home to one of the most dynamic life sciences clusters in the United States, with more than 600 life science companies, 75 percent in the Research Triangle, according to the Life Sciences 2020 report from Cushman and Wakefield,

Cushman and Wakefields numbers differ from those cited by the NC Biotech Center, which says there are more than 735 life sciences companies in the state.

Among other metrics cited: life sciences companies employ 6.9 thousand employees, up 84 percent since 2010; a 10.7 percent vacancy for office/lab space; the $20.1 billion in National Institute of Health funding for UNC-CH, $2 billion at Duke; and $160 million for the Research Triangle Institute. NC State, which also lands significant NIH funding, is not listed.

The report notes that Research Triangle Park is centrally located between Raleigh and Durham and is the largest research park in North America. It is home to industry giants such as GlaxoSmithKline, IQVIA, PPD, and Biogen. It also includes university spinouts and startups among Fortune 100 international corporations on its sprawling 7,000 acre campus.

The report points out that Forbes ranked NC as number one among the best states for business in 2017 and 2018. Key factors, it says, are extremely low cost of doing business, minimal regulations, impressive growth statistics, and a high quality of life.

Most notably, the report states, is home to three separate research universities (Duke, the University of North Carolina at Chapel Hill, and NC State University) which graduate 53,000 students annually. This provides life science companies with a pool of top talent.

The report lists top life science companies, top venture-funded companies, inventory growth projections, notable lease transactions and notable real estate sales transactions.

Other biotech hubs examined in the report are: Baltimore, Boston, Chicago, Montreal, New York City, New Jersey, Oakland/East Bay, Philadelphia, San Diego, San Francisco, Seattle, Toronto, and Washington DC.

Doug Edgeton, president and CEO of the NC Biotech Center, had this response to the report:

Were always pleased to be among the nations leaders in the annual Cushman & Wakefield life science report. It is, of course, quite specific in its focus on real estate, its always useful to see how we compare with other major life science hubs.

It also serves as a reminder of the importance of some recently announced projects that arent reflected in this survey, including Eli Lillys January announcement of a $474 million, 462-employee pharma manufacturing facility its building in RTP Lillys first in the state.

NC Biotechnology Center CEO Doug Edgeton

That followed Mercks announcement of a $680 million, 425-employee vaccine production expansion thatll add 225,000 square feet to its existing and already impressive Durham campus.

Fresenius Kabi is adding a $100 million 445-employee expansion of its pharmaceutical factory in Wilson. And Seqirus is constantly expanding its huge cell-based flu vaccine factory in Holly Springs, where its about to occupy a $140 million, 120-employee addition to give it 475,000 square feet of space the size of eight football fields at its 185-acre site.

And Pfizer has just embarked on a build-out of a $600 million, 340-employee gene therapy research and manufacturing facility at its 230-acre campus in Sanford, plus a $19 million investment in a 60,000-square-foot building on a 16-acre site in Durham. Pfizer already employs about 3,600 people in North Carolina at sites in Chapel Hill, Sanford and Morrisville.

And there are more announcements on the near horizon, because North Carolina is a great place to do business, especially in the life sciences. In fact, Forbes named us the best state for business in both 2017 and 2018.

Those same years, Site Selection Magazine has named us the top competitive state. And just last month The Boyd Company released a study comparing the top 25 global biopharma hubs for cost of operating a biomanufacturing plant.

We came in at number three, beaten only by Bangalore, India and Tel Aviv, Israel. We beat Austin, Texas; Denver; Pittsburgh; St. Louis; Seattle; Montgomery County, Maryland; and of course, San Diego, Philadelphia and Chicago.

We can never become complacent, because the life science world is extremely competitive, very rewarding, and necessary for global health and well-being. And were glad to be among the leaders of the pack.

Excerpt from:
Report says: North Carolina home to one of the most dynamic life science clusters in the US - WRAL Tech Wire

Recommendation and review posted by Bethany Smith

An emergency room physician, initially unable to diagnose a disoriented patient, finds on the patient a wallet-sized card providing access to his genome, or all his DNA. The physician quickly searches the genome, diagnoses the problem and sends the patient off for a gene-therapy cure. Thats what a Pulitzer prize-winningjournalist imagined2020 would look like when she reported on the Human Genome Project back in 1996.

The Human Genome Project was an international scientific collaboration that successfully mapped, sequenced and made publicly available the genetic content of human chromosomes or all human DNA. Taking place between 1990 and 2003, the project caused many to speculate about the future of medicine. In 1996, Walter Gilbert, a Nobel laureate,said, The results of the Human Genome Project will produce a tremendous shift in the way we can do medicine and attack problems of human disease. In 2000, Francis Collins, then head of the HGP at the National Institutes of Health,predicted, Perhaps in another 15 or 20 years, you will see a complete transformation in therapeutic medicine. The same year, President Bill Clintonstatedthe Human Genome Project would revolutionize the diagnosis, prevention and treatment of most, if not all, human diseases.

It is now 2020 and no one carries a genome card. Physicians typically do not examine your DNA to diagnose or treat you. Why not? As I explain in a recentarticle in the Journal of Neurogenetics, the causes of common debilitating diseases are complex, so they typically are not amenable to simple genetic treatments, despite the hope and hype to the contrary.

The idea that a single gene can cause common diseases has been around for several decades. In the late 1980s and early 1990s, high-profile scientific journals, including Nature and JAMA, announced single-gene causation ofbipolar disorder,schizophreniaandalcoholism, among other conditions and behaviors. These articles drewmassive attentionin thepopular media, but weresoonretractedorfailedattemptsatreplication. These reevaluations completely undermined the initial conclusions, which often had relied onmisguided statistical tests. Biologists were generally aware of these developments, though the follow-up studies received little attention in popular media.

There are indeed individual gene mutations that cause devastating disorders, such asHuntingtons disease. But most common debilitating diseases are not caused by a mutation of a single gene. This is because people who have a debilitating genetic disease, on average, do not survive long enough to have numerous healthy children. In other words, there is strong evolutionary pressure against such mutations. Huntingtons disease is an exception that endures because it typically does not produce symptoms until a patient is beyond their reproductive years. Although new mutations for many other disabling conditions occur by chance, they dont become frequent in the population.

Instead, most common debilitating diseases are caused by combinations of mutations in many genes, each having a very small effect. They interact with one another and with environmental factors, modifying the production of proteins from genes. The many kinds of microbes that live within the human body can play a role, too.

A silver bullet genetic fix is still elusive for most diseases. (Credit: drpnncpptak/Shutterstock)

Since common serious diseases are rarely caused by single-gene mutations, they cannot be cured by replacing the mutated gene with a normal copy, the premise for gene therapy.Gene therapyhas gradually progressed in research along a very bumpy path, which has included accidentally causingleukemiaandat least one death, but doctors recently have been successful treatingsome rare diseasesin which a single-gene mutation has had a large effect. Gene therapy for rare single-gene disorders is likely to succeed, but must be tailored to each individual condition. The enormous cost and the relatively small number of patients who can be helped by such a treatment may create insurmountable financial barriers in these cases. For many diseases, gene therapy may never be useful.

The Human Genome Project has had an enormous impact on almost every field of biological research, by spurring technical advances that facilitate fast, precise and relatively inexpensive sequencing and manipulation of DNA. But these advances in research methods have not led to dramatic improvements in treatment of common debilitating diseases.

Although you cannot bring your genome card to your next doctors appointment, perhaps you can bring a more nuanced understanding of the relationship between genes and disease. A more accurate understanding of disease causation may insulate patients against unrealistic stories and false promises.

This article is republished from The Conversation under a Creative Commons license. Read the original article. This opinions expressed in this article belong solely to the author.

See the original post:
We've Sequenced the Human Genome. So Why Haven't We Cured More Diseases? - Discover Magazine

Recommendation and review posted by Bethany Smith

IRVINE, Calif., Feb. 14, 2020 (GLOBE NEWSWIRE) -- ClearPoint Neuro, Inc. (Nasdaq: CLPT) (the Company), a leading platform neurosurgery company, today announced a strategic agreement with PTC Therapeutics, Inc. (PTC). The scope of the agreement includes hardware, software, clinical case and market development services for gene therapy cases in the United States and Europe to support PTCs potential commercialization in gene therapy globally upon regulatory approval. In addition to the announcement of this agreement, ClearPoint Neuro today announces the following 2020 Outlook for the companys performance:

We have put a tremendous amount of thought and effort into redefining our Company over the past two years, commented Joe Burnett, President and CEO of ClearPoint Neuro, Inc. This has included the vision, the team, the partnerships, and even the name of our company which officially changed this week to ClearPoint Neuro from MRI Interventions. We are thrilled by the evolution our Company has already undergone and believe we have only scratched the surface of the significant potential ahead.

We have evolved to become two companies in one, continued Burnett. On one side, we are a platform medical device company, consistently delivering double digit growth, and continuing to expand our installed base of neurosurgery centers in the U.S. Every year more surgeons and more patients gain access to the ClearPoint system and compatible disposable devices. On the other side of the business, we are a gene therapy and biologics enabling company, providing navigation, drug delivery, and case support to more than 20 companies in this exciting and growing space. Here we currently support pre-clinical and clinical efforts, but we believe that we are on the precipice of potentially explosive growth as these therapies progress through the regulatory process toward commercial launch. We feel that our company represents both scale and purpose through a unique combination of predictable device growth and a potential biologics opportunity, all supported under a common team dedicated to treating the most debilitating neurological disorders.

ClearPoint Neuros revenue outlook for 2020 includes an expectation that approximately 33% of total revenue will be derived from biologics and drug delivery products and services, up from approximately 20% in 2019, and reflecting the growth of this part of the business.

When planning for the development and potential commercialization of gene therapy globally, safety, consistency and predictability are crucial constructs that all must be included, commented Marcio Souza, Chief Operating Officer of PTC Therapeutics and ClearPoint Neuro Board Member. Our agreement with ClearPoint Neuro is designed to provide standardization across all of our centers of excellence as we can maintain consistency in navigation, delivery and clinical support. The more variables we can control, the more successful we believe the outcomes for patients will be.

As part of our Companys duality in devices and biologics, we are thrilled to deepen our partnership with PTC, commented Jacqueline Keller, Vice President and Program Manager at ClearPoint Neuro for the PTC Partnership. This new agreement with PTC highlights a turn-key solution for our gene therapy partners, whereby our commercial organization, with deep relationships in the neurosurgery community, can take on the burden of sales and clinical activities in the surgical suite, and allow our partners who are commonly small-molecule focused companies to continue to prioritize their efforts with neurologists, patients and reimbursement administrators. With the capacity to support thousands of cases each year, our team plans to provide this service across multiple companies and indications in neuro.

About the Company

The Companys mission is to improve and restore quality of life to patients and their families by enabling therapies for the most complex neurological disorders with pinpoint accuracy. Applications of the Companys current product portfolio include deep-brain stimulation, laser ablation, biopsy, neuro-aspiration, and delivery of drugs, biologics and gene therapy to the brain. The ClearPoint Neuro Navigation System has FDA clearance, is CE-marked, and installed in 60 active clinical sites in the United States. The Companys SmartFlow cannula is being used in partnership or evaluation with more than 20 individual biologics and drug delivery companies in various stages from preclinical research to late stage regulatory trials. To date, more than 3,500 cases have been performed and supported by the Companys field-based clinical specialist team which offers support and services for our partners. For more information, please visit http://www.clearpointneuro.com.

Note on Forward-Looking Statements

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995 regarding the planned offering. Additionally, all statements relating to any closing(s) of, and the amount or use of any proceeds from, the transactions described in this press release are considered to be forward-looking statements. Other forward-looking statements may be identified by the words guidance, plan, anticipate, believe, estimate, expect, intend, may, target, potential, will, would, could, should, continue, and similar expressions. Forward-looking statements are subject to risks and uncertainties, and the Companys actual results and the timing of events could differ materially from those anticipated in such forward-looking statements as a result of such risks and uncertainties, which include, without limitation, risks and uncertainties associated with market conditions and the satisfaction of closing conditions related to the transactions described in this press release. There can be no assurance that the parties will be able to complete the transactions described in this press release on the terms described herein or in a timely manner, if at all. More detailed information on these and additional factors that could affect the Companys actual results are described in the Risk Factors section of the Companys Annual Report on Form 10-K for the year ended December 31, 2018, and the Companys Quarterly Reports on Form 10-Q for the periods ended March 31, 2019, June 30, 2019 and September 30, 2019, all of which have been filed with the SEC, as well as the Companys Annual Report on Form 10-K for the year ended December 31, 2019, which the Company intends to file with the Securities and Exchange Commission on or before March 30, 2020. You are urged to carefully consider all such factors. Copies of these and other documents are available from the Company. The forward-looking statements contained herein represent the Companys views only as of the date of this press release the Company does not undertake or plan to update or revise any such forward-looking statements to reflect actual results or changes in plans, prospects, assumptions, estimates or projections, or other circumstances occurring after the date of this press release except as required by law.

For More Information

ClearPoint Neuro, Inc.Matt KrepsDarrow Associates Investor Relations(214) 597-8200mkreps@darrowir.com

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ClearPoint Neuro, Inc. Announces 2020 Revenue Outlook Ahead of Nasdaq Investor Presentation TodayNew US and European Partnership Agreement with PTC...

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MUNICH--(BUSINESS WIRE)--SIRION Biotech GmbH (SIRION), a world leader in viral vector-based gene delivery technologies for gene and cell therapy, today announced preliminary financial results for 2019. SIRIONs annual service and licensing revenues in 2019 exceeded 10M (US$11.2M). With a growing staff of 35 employees, the company reports a record profit and plans to invest a portion of these proceeds into an additional Munich site with expanded lab and process development capacities. In addition, SIRIONs offices in Paris and Boston will continue to expand.

In addition, the companys intellectual property is included in more than 10 clinical programs by leading drug developers, one of which was granted market approval in 2019. SIRIONs core fee-for-service business, providing vector materials for discovery and pre-clinical applications, experienced historic growth of more than 50% over the previous year. The company expanded into a new avenue of growth by assigning its share in joint intellectual property rights to both a client in USA and the Danish start-up, InProTher Aps, in return for shares and milestone payments as well as royalties on product net sales.

SIRION Biotechs business model comes from service revenue and licensing fees. The company provides high-grade viral vector materials for preclinical use, offering up to 1015 GMP-ready AAV vector genomes and 1010 infectious units of Lentivirus, currently paving all the way to toxicology studies. In addition, the company is forecasting significant increased demand for its clinical and commercial grade vectors.

As SIRION continues its growth trajectory, it will focus on attracting funding to expand preclinical development with collaborators from its large client network. With more than 2,000 single projects for over 200 recurring clients worldwide, SIRION is committed to building significant intelligence and insights into latest gene therapy drug developments.

After only 12 years in business, SIRION has been instrumental in bringing new gene therapy treatments to patients. Assigning intellectual properties to collaborators validates our comprehensive viral vector technology platforms and the highly skilled and creative lab staff that stands behind them. We continue our dedication and commitment to developing gene therapy on a global scale with the opening of our second Munich site this year that will expand our capacity for research and process development, said Dieter Lingelbach, Chief Operating Officer of SIRION.

Mr. Lingelbach continued, In 2020, we look forward to continued expansion of our US presence through our wholly-owned subsidiary, SIRION Biotech International Inc., as well as continued growth in Paris.

Gene and cell therapies are among the hottest topics in modern drug development. Viral vector technologies are the most promising gene editing tools available today, with significant potential both as therapeutics for a growing number of indications, for tumor vaccines and for further technical improvements. Key challenges include quality, yields, and improved transduction in order to make novel gene therapies reliable and affordable. Costs for gene therapies per patient remain high, and can be prohibitive for larger patient populations, even in well-developed markets.

About SIRION Biotech GmbHSIRION Biotech was founded in 2005 to lead the next generation of viral vector technologies for gene and cell therapy as well as vaccine development. Now SIRION offers one of the worlds most comprehensive viral vector technology platforms based on lenti-, adeno-, and adeno-associated viruses which expedites gene therapy research and advances drug development. SIRION is becoming a partner of choice in this growing sector. LentiBOOST has been used in several clinical trials from early stage clinical Phase 1/2 through late stage clinical Phase 3 trials and demonstrated clinical success in improving transduction of the therapeutic vector.

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Gene and Cell Therapy Solutions Provider, SIRION Biotech Reports Record Growth for Year Ended 2019 Based on Preliminary Financial Results - Business...

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In pulmonary arterial hypertension (PAH), high blood pressure in the lungs arteries causes the heart to work extra hard to pump blood to the lungs and around the rest of the body. The condition is rare but deadly, and current treatments are expensive and have side effects and inconvenient modes of delivery. There is no cure.

With a goal of developing a more effective, convenient, and affordable therapy, research led by Henry Daniell of Penns School of Dental Medicine produced a protein drug in lettuce leaves to treat PAH. He worked with other scientists, including Steven M. Kawut of Penns Perelman School of Medicine; Tim Lahm from the Indiana University School of Medicine; Maria Arolfo and Hanna Ng of the Stanford Research Institute, on toxicology and pharmacokinetic studies; and Cindy McClintock and Diana Severynse-Stevens of RTI International, on regulatory studies.

The protein drug, composed of the enzyme angiotensin converting enzyme-2 (ACE2) and its protein product angiotensin (1-7), can be taken orally and, in an animal model of PAH, reduced pulmonary artery pressure and remodeling. In addition, rigorous toxicology and dose-response studies suggested the drugs safety in animals. Further work will be necessary to develop this novel treatment approach for patients with PAH. The teams findings appear in the March issue of the journal Biomaterials.

We completed extensive investigations to highly express these proteins in lettuce plants and to ensure the product is safe and effective, says Daniell. Were ready to progress with further work to move this to the clinic.

Daniell has employed his innovative platform to grow biomedically important proteins of many kinds in the leaves of plants. The system works by physically bombarding plant tissue with the genes of interest, prompting chloroplasts into taking up genes and then stably expressing that protein. Propagating those plants then creates a kind of pharmaceutical farm from which the researchers can harvest, dry, and process the leaves, resulting in a powder that can be placed in a capsule or suspended in a liquid for use as an oral medication.

A 2014 publication in the journal Hypertension, on which the current study was based, earned Daniell a prize from the American Heart Association, and support from the National Institutes of Health through its Science Moving TowArds Research Translation and Therapy (SMARTT) program, which aims to efficiently translate promising basic science discoveries into therapies that can make a difference in peoples lives.

That earlier publication had shown that ACE2 and angiotensin (1-7) could be expressed in tobacco leaves and, when fed to rats with a condition that models pulmonary arterial hypertension, could significantly reduce the animals pulmonary artery pressure while also improving cardiac function.

To create a drug that humans could safely ingest, however, required moving from a tobacco to a lettuce-based platform. The new work takes advantage of other advancements the Daniell lab has made during the last several years. He and colleagues have successfully devised methods to enhance expression of human genes in the plants and to remove the antibiotic resistance gene that is used to select for angiotensin-producing plants. Theyve also worked with a partner to produce genetically engineered plants in a production facility that adheres to FDA standards.

In the current work, the researchers demonstrated that they could accurately evaluate the dose of the ACE2 and angiotensin (1-7) proteins in lettuce, and that the products could be dried and kept shelf stable for as long as two years.

Funding from the SMARTT program enabled animal studies evaluating toxicology, pharmacodynamic, and pharmacokinetic studies, which evaluate the safety of the drug, where it goes in the body, and how long it persists in the body at different doses, in work done at Stanford University.

And to confirm that the lettuce formulation of the product had a positive impact on experimental PAH, the team fed rats a solution containing the drug for four weeks. Their lung pressures went down 30-50%, and the structure of their arteries also improved.

This is an innovative approach to targeting the renin-angiotensin-aldosterone system in pulmonary arterial hypertension, says Penn Medicines Kawut, which may hold promise in this and other diseases.

We are very excited about this work that shows efficacy of bioencapsulated ACE2 and angiotensin (1-7) in our animal model of pulmonary arterial hypertension, says Indiana Universitys Lahm. We now need to confirm that the intervention also works in other animal models and when given later in the disease. Ultimately, our goal is to move this to the clinic for trials in patients, but we need to make sure we learn as much as possible from animal studies and from studies in healthy human subjects to make sure this intervention is safe and efficacious in patients.

In other future work, Daniell hopes to continue evaluating the effects of ACE2 and angiotensin (1-7) in treating different types of cardiovascular disease, such as heart failure.

There are some potentially broad applications of this drug that were hoping to investigate, says Daniell.

Daniell, Kawut, and Lahms coauthors on the paper were Penn Dental Medicines Venkata Mangu, Jiyoung Park, Peyman Habibi, Yao Shi, and Patricia A. Gonnella; and Indiana Universitys Bakhtiyor Yakubov, Amanda Fisher, Todd Cook, and Lily Zeng.

Henry Daniell is vice chair and W.D. Miller Professor in the Department of Basic & Translational Sciences in the University of Pennsylvania School of Dental Medicine.

Steven M. Kawut is professor of medicine and director of the Pulmonary Vascular Disease Program at the University of Pennsylvania Perelman School of Medicine.

Funding for the study came from the National Institutes of Health (NIH) (grants HL107904, HL109442, and HL133191) and through the NIHs Science Moving TowArds Research Translation and Therapy (SMARTT) program (contracts HHSN268201600011C and HHSN268201600014C).

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Advancing an oral drug for pulmonary arterial hypertension - Penn: Office of University Communications

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New research has identified gene variants found only in the brains of transgender individuals. These genes are primarily involved in estrogens critical sprinkling of the brain right before or after birth, which is essential to masculinization of the brain.

The study, which included 13 transgender males (female to male) and 17 transgender females (male to female), reveals some of the first biological evidence of the incongruence transgender people experience because their brain indicates they are one sex and their body another.

Twenty-one variants in 19 genes have been found in estrogen signaling pathways of the brain critical to establishing whether the brain is masculine or feminine, says Dr. J. Graham Theisen, obstetrician/gynecologist and National Institutes of Health Womens Reproductive Health Research Scholar at the Medical College of Georgia at Augusta University.

In natal males (people whose birth sex is male), this critical estrogen exposure doesnt happen, or the pathway is altered so the brain does not get masculinized. In natal females, it may mean that estrogen exposure happens when it normally wouldnt, leading to masculinization.

Both could result in an incongruence between a persons internal gender and their external sex. The negative emotional experience associated with this incongruence is called gender dysphoria.

They are experiencing dysphoria because the gender they feel on the inside does not match their external sex, Theisen says. Once someone has a male or female brain, they have it and you are not going to change it. The goal of treatments like hormone therapy and surgery is to help their body more closely match where their brain already is.

The findings are published in the journal Scientific Reports.

It doesnt matter which sex organs you have, its whether estrogen, or androgen, which is converted to estrogen in the brain, masculinizes the brain during this critical period, says Dr. Lawrence C. Layman, chief of the MCG Section of Reproductive Endocrinology, Infertility and Genetics in the Department of Obstetrics and Gynecology. We have found variants in genes that are important in some of these different areas of the brain.

While its too early to definitively say the gene variants in these pathways result in the brain-body incongruence called gender dysphoria, it is interesting that they are in pathways of hormone involvement in the brain and whether it gets exposed to estrogen or not, says Layman.

This is the first study to lay out this framework of sex-specific development as a means to better understand gender identity, Theisen says. We are saying that looking into these pathways is the approach we are going to be taking in the years ahead to explore the genetic contribution to gender dysphoria in humans.

For the study, the team looked at the DNA of 13 transgender males, individuals born female and transitioning to male, and 17 transgender females, born male and transitioning to female.

The extensive whole exome analysis was conducted at the Yale Center for Genome Analysis.The variants found were not present in a group of 88 control exome studies in nontransgender individuals also done at Yale. They also were rare or absent in large control DNA databases.

Reproductive endocrinologist/geneticist Layman says his 20 years of experience taking care of transgender patients made him think there was a biological basis. We certainly think that for the majority of people who are experiencing gender dysphoria there is a biologic component, says Theisen. We want to understand what the genetic component of gender identity is.

Although genetics have been suggested as a factor in gender dysphoria, proposed candidate genes to date have not been verified, the researchers say. Most gene or gene variants previously investigated have been linked to receptors for androgens, hormones more traditionally thought to play a role in male traits but, like estrogen in males, are also present in females.

The team decided instead to take what little is known about sex-specific brain development that estrogen bath needed in early life to ensure masculinization of the brain to hone in on potential sites for relevant genetic variances.

Extensive DNA testing initially revealed more than 120,000 variants, 21 of which were associated with these estrogen-associated pathways in the brain.

Theisen notes that we all are full of genetic variants, including ones that give us blue eyes versus brown or green, and the majority do not cause disease, but rather help make us individuals. I think gender is as unique and as varied as every other trait that we have, Theisen says.

The investigators suggest modification of the current system for classifying variants that would not imply that a variant means pathogenic (disease causing).

Last year, the World Health Organization said that gender incongruence is not a mental health disorder and six years before that, the Diagnostic and Statistical Manual of Mental Disorders replaced gender identity disorder with general dysphoria.

About 0.5 to 1.4% of individuals born male and 0.2 to 0.3% of individuals born female meet criteria for gender dysphoria. Identical twins are more likely than fraternal twins to both report gender dysphoria.

Gender affirming therapies, like hormone therapies and surgeries, along with mental health evaluation and support help these individuals better align their bodies and brains, the scientists say.

Source: Medical College of Georgia at Augusta University

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Study IDs Gene Variants Potentially Linked to Brain-Body Incongruence in Transgender - PsychCentral.com

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Maybe were all just batteries. Thats the bad news for Keanu Reeves Neo, who wakes up from a chemically-induced slumber and discovers hes nothing more than a pack of double-As connected to a massive power station run by evil robot overlords in the dystopian sci-fi film The Matrix.

But what if the power potential of human electric current wasnt so post-apocalyptic? As the International Energy Agency notes, global demand for power rose 2.3% in 2018, which represents the biggest increase in the last decade. Ever since Ben Franklin first decided that flying kites in dangerous weather was solid scientific practice, humans have been finding new ways to use electricity and discovering that demand never stops.

The bad news? Youre no coppertop. The better news? Bioelectricity is essential to life and may drive the future of human development.

Harvesting electricity from human activity is nothing new. As Knowable Magazine notes, breathing can produce more than 0.80 watts, body heat can generate up to 4.8 watts, and the motion of your arms creates a stunning 60 watts of power.

But the notion of electricity as fundamental to biological life isnt quite so clear-cut. According to Quartz, while there were some experiments measuring human electrical currents in the mid-1920s, its wasnt until 1949 that Alan Hodgkin and Andrew Huxley identified the movement of ions across cell nerve membranes. The pair took home a Nobel Prize for their work, but this electric revolution was quickly outpaced by the double-helix discovery of DNA. For decades, genes became the best-fit scientific foundation for biology, while electricity research was short-circuited.

Then, in 1976, Erwin Neher and Bert Sakmann developed a tool capable of circumventing bioelectricitys biggest problem: studying ion movements without killing their cellular transport mediums. And later, in 2012, Richard Nuccitelli created a device sensitive and subtle enough to track human electric currents on skin, and discovered that, when skin cells are wounded, they emit an injury current that calls for help from other cells. The larger the wound, the bigger the current and the current decreases with age. Other work found that charges inside embryo cells significantly affected development. As NOVA states, Researchers overwhelmingly agree that bioelectric currents are essential to nerve and muscle function.

With the human nervous system constantly generating a fluctuating electric current, why cant we all just plug in and power up? It all comes down to the two halves of electric potential: positive and negative charges.

The electricity were most familiar with the kind Franklin flew kites for and that powers our smartphones, dishwashers and light bulbs depends on the flow of negatively-charged electrons to produce a current. Meanwhile, in our bodies, its the movement of positively-charged ions such as potassium, sodium and calcium passing through cell membranes that create electric potential. And while this variable voltage is essential to keep hearts beating, limbs moving and minds functioning, its not great for typical electrical applications. For example, when animal cells take in sodium and chloride ions and discharge potassium ions, the result is a voltage between -40 to -80 mV across membranes, significantly less than a single watch battery.

However, as it turns out, human electric current offers significant potential for internal applications.

The biggest potential for human-produced power? Improved healing. Studies published in the journal Advances in Wound Care have shown that supplementing the bodys electric current with outside electrical stimulation can help to reduce the recovery time needed for bedsores, which are some of the most difficult wounds to mitigate, let alone fully heal. Similar work has shown improvements in healing bone fractures.

Next on the list? Cancer. While research in the 1920s demonstrated a connection between changing electric gradients and cancerous tumors, cell mutations are the most commonly cited cause of cancer concerns. Now, theres speculation that misregulation of electric currents may lead to cellular communication challenges in effect, cells forget theyre part of a larger network and begin acting selfishly by hoarding resources and growing out of control. Research from the University of Nottingham found that biologically-generated currents underpin specific cancer cell behaviors, and new techniques using a combination of gene therapy and light-activated ion channels have seen success treating cancer in tadpoles.

However, despite steady progress, challenges remain. Human genomes are far more complex than those of rats or tadpoles, and gene therapies face significant regulatory challenges. Electric treatments for wound healing also struggle with standardization how long should currents be applied to wounds for maximum effect without causing secondary damage? At what voltage? Using what type of device? Is alternating current (AC) or direct current (DC) safer? More effective?

The result is a kind of cautious optimism. While bioelectric benefits are grounded in solid science, more testing and research is necessary to standardize and streamline medical processes.

Bioelectric research offers the tantalizing potential to tap the inherent power of the human nervous system, but were not there yet.

Still, theres good reason to be optimistic. Mike Levin of Tufts University, whose lab is on the leading edge of human electric current research, puts it simply: Understanding the bioelectricity, biomechanics, and transcriptional circuits that allow cells to cooperate toward large-scale goals is the key to regenerative medicine, birth defects, cancer reprogramming, aging, synthetic bioengineering, and even new AI.

Put simply? Were not batteries thanks to positive bioelectric potential, were even better.

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Feb 14, 2020 (Thomson StreetEvents) -- Edited Transcript of Urovant Sciences Ltd earnings conference call or presentation Thursday, February 13, 2020 at 9:30:00pm GMT

Urovant Sciences Ltd. - CFO & Senior VP of Business Development

Urovant Sciences Ltd. - Chief Medical Officer of USI

* Keith A. Katkin

Urovant Sciences Ltd. - CEO & Director

Urovant Sciences Ltd. - Executive Director of IR

* Biren N. Amin

H.C. Wainwright & Co, LLC, Research Division - Equity Research Associate

Good day, ladies and gentlemen, and welcome to Urovant Fiscal Third Quarter 2019 Earnings Call.

(Operator Instructions) As a reminder, today's conference is being recorded. I would now like to turn the call over to Ryan Kubota. Sir, you may begin.

Ryan Kubota, Urovant Sciences Ltd. - Executive Director of IR [2]

Thank you, Fati. Good afternoon, and thank you all for joining Urovant's Fiscal 2019 Third Quarter Financial Results Conference Call.

With me today are 4 members of our leadership team. Keith Katkin, Chief Executive Officer; Ajay Bansal, Chief Financial Officer; Dr. Cornelia Haag-Molkenteller, Chief Medical Officer; and Christine Ocampo, Chief Accounting Officer.

Today, after market close, we issued a press release containing detailed information on our quarterly results. You may access our release on our company website, urovant.com.

During our call, we'll be making forward-looking statements, including statements regarding our plans and strategies for the clinical development of vibegron and other treatments for urologic diseases.

Listeners are cautioned that all of our forward-looking statements are based on our current expectations and assumptions, which are subject to numerous risk factors that could cause our actual results to differ materially. Accordingly, we advise listeners to review the forward-looking statements disclosure in today's press release and the Risk Factors section of our Form 10-K, as well as our Form 10-Q, which we filed later today.

With that said, I will now turn the call over to our CEO, Keith Katkin. Keith?

Keith A. Katkin, Urovant Sciences Ltd. - CEO & Director [3]

Thank you, Ryan, and my thanks for all of you for joining us this afternoon.

This has been a very exciting and transformational quarter for Urovant, marked by key milestones across all aspects of our business.

First, in December, we submitted our New Drug Application to the U.S. Food and Drug Administration for vibegron for the treatment of patients with overactive bladder. The submission comes one quarter earlier than we initially anticipated, and we are very excited to have submitted our application by the end of the calendar year and ahead of schedule.

Second, we initiated our Phase IIa study in URO-902, our novel gene therapy product for patients with overactive bladder, suffering with urge urinary incontinence that have failed oral pharmacologic therapy. The study is evaluating URO-902's efficacy, safety and tolerability of a single administration of the product, and we expect top line safety data towards the end of the year.

Third, pursuant to the transaction between Sumitomo Dainippon Pharma, also known as DSP, and Roivant Sciences, DSP is now Urovant's majority shareholder. As part of the transaction, Urovant entered into a loan agreement with DSP, whereby DSP provided Urovant with a $300 million low interest, interest-only 5-year term loan facility with no repayments due until the end of the term. This loan facility provides Urovant with capital well into 2021 and enabled us to pay back the outstanding amount on our previous loan agreement with Hercules Capital.

DSP also expects to support Urovant through profitability and provide support for the commercialization of vibegron, providing access to its U.S. commercial infrastructure, including drug distribution, operations and managed care support.

Finally, Urovant and DSP entered into an investor rights agreement that provides certain protections to Urovant minority shareholders for as long as DSP holds between 50% and 90% of Urovant's outstanding voting power.

We're excited about our new strategic relationship with DSP, the potential benefits that it brings to Urovant and the future of our company. With the submission of our New Drug Application and launch preparations for vibegron well underway, we look forward to the strategic support and proven commercial resources of DSP.

Their drug distribution, operational and managed care support capabilities should greatly optimize Urovant's commercial approach to the U.S. market.

Now to comment briefly on some of our other important business highlights. We continue to enroll patients into Part 2 of the Phase III COURAGE trial, which will assess both efficacy and safety of vibegron in men with OAB and BPH, a patient population for which no product is currently approved.

Enrollment also continues for patients into our IBS-associated abdominal pain trial with top line results expected later this year.

With that, I'll now turn the call over to our Chief Medical Officer, Dr. Cornelia Haag-Molkenteller, who'll provide more detail on our clinical programs.

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Cornelia Haag-Molkenteller, Urovant Sciences Ltd. - Chief Medical Officer of USI [4]

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Thank you, Keith. As mentioned before, we are pleased to have submitted our new drug application for vibegron for the treatment of OAB to the FDA at the end of last year.

Let me briefly outline what to expect regarding the OAB filing and the program, then provide an update on our other clinical development programs.

In OAB, first, the submission of a New Drug Application to the FDA at the end of December 2019, we'll be waiting for the notification by the FDA of the acceptance of our file in March of 2020.

Second, we're expecting a 12-month review cycle at the FDA and potential approval could occur at the end of this year. We are looking forward to working with the FDA during the review process.

Third, in May, at the upcoming AUA, American Urological Association's 2020 Annual Meeting in Washington D.C., we will have 2 presentations: the first presentation would be our 52-week EMPOWUR extension study data; and the second presentation will be that of the EMPOWUR data by age.

Regarding the EMPOWUR extension study, we performed a post hoc analysis to test the difference in week -- at week 52 between vibegron 75 milligram and the active comparator tolterodine on the change of baseline in the urge urinary incontinence and total incontinence episodes between vibegron and tolterodine.

Notably, for both parameters, vibegron demonstrated a statistically significant reduction over tolterodine. The symptoms of urinary incontinence are key symptoms OAB and the main reason for patients to seek treatment. We're very excited by this data and believe this further supports the strong efficacy profile of vibegron.

Let me now turn to our other clinical programs. I'm pleased to report that we continue to make excellent progress across all of our development programs. Regarding our international, Phase III COURAGE development program for vibegron in men with OAB and benign prostatic hyperplasia, or BPH, we continue to enroll patients into Part 2 of the trial, which over 1,000 patients will be enrolled.

The trial is running in North America, and we will soon be initiating studies in Europe.

Our 2 of the Phase III current trail, which will assess both the efficacy and safety of vibegron in men with OAB and BPH. The co-primary endpoints are reduction in micturition frequency and urgency episodes per 24 hours. Key secondary endpoints are reduction in nocturia episodes, which means the awakening at night avoid, prostate symptom scores and safety. In addition, the first patients from Part I have enrolled into the long-term extension study, which will follow patients for a total exposure of 52 weeks.

We're excited about this program as there is currently no FDA-approved product specifically indicated for overactive bladder in men with BPH.

Let me now turn to our Phase IIa clinical program for IBS-associated abdominal pain. The study continues to enroll female patients with IBS-associated pain. Patients are randomized to either 75-milligram of vibegron or placebo. The primary endpoint is a 30% reduction in abdominal pain intensity on an 11-point rating scale over 12 weeks for IBS-D, which is the IBS with diarrhea. A responder is defined with the subject with at least 30% decrease in worst abdominal pain compared to the weekly baseline average.

Secondary endpoints include a global rating scale and safety, in particular, a lack of negative effects of stool frequency or consistency. We expect to report top line data from this study in the third quarter of 2020.

Finally, as reported recently, we initiated a Phase IIa trial for URO-902 in December 2019, our novel injectable gene therapy for patients with OAB, who've failed oral pharmacologic therapy.

This is a randomized, double-blind, placebo-controlled trial that will evaluate the efficacy, safety and tolerability of a single administration of URO-902. The therapy is administered by direct intradetrusor injections into the bladder wall under local anesthesia.

The trial is expected to enroll approximately 80 female patients in the U.S., 2 cohorts. The first cohort will receive either single administration of 24 milligram of URO-902 or matching placebo. The second cohort will receive 48 milligrams of URO-902 or matching placebo. Patients will be then followed for 48 weeks.

The primary outcome measure is the change in the average daily number of urge urinary incontinence for baseline, 2 week trial as well as assessment of safety and tolerability for this potential new therapy.

I am pleased with the progress we made over the last quarter and look forward to providing you with further updates in the coming quarters.

Now I'll pass to Ajay for a financial update.

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Ajay Bansal, Urovant Sciences Ltd. - CFO & Senior VP of Business Development [5]

--------------------------------------------------------------------------------

Thank you, Cornelia.

In addition to the financial results summarized in our press release, you can find additional information in our upcoming Form 10-Q, which will be filed later today.

R&D expenses were $23.1 million for the third quarter of 2019 compared with $21.3 million for the same period in the prior year. In the third quarter of 2019, R&D expenses were comprised of costs related to the submission of a new drug application for vibegron to the U.S. FDA.

Clinical development. Specifically, our ongoing studies in OAB plus BPH and abdominal pain associated with IBS. When comparing the 2 quarters, the increase in R&D expenses is primarily due to: a $2.9 million PDUFA fee for our NDA submission of vibegron for the treatment of OAB; a $2.5 million milestone payment as part of a collaboration agreement to Kyorin Pharmaceuticals in connection with our FDA submission of vibegron; and an increase of $2.6 million in share-based compensation due to the acceleration of vesting of certain stock options and RSUs as a result of the sale of Roivant's interest in the company to DSP.

These increased expenses were partially offset by lower clinical research organization costs, primarily due to the completion of Phase III EMPOWUR study earlier this fiscal year.

G&A expenses were $16.7 million for the third quarter of 2019 compared with $4.9 million for the same period in the prior year. The increase in G&A expense is primarily due to an increase of $8.7 million in share-based compensation from acceleration of vesting of certain stock options and RSUs and an increase in personnel costs as well as other general and corporate expenses.

Total operating expenses were $39.8 million for the third quarter of 2019 compared with $26.2 million for the same period in the prior year. As mentioned before, the increase is primarily driven by the increase in share-based compensation expense, PDUFA fee and Kyorin milestone payment.

Cash used in operations was $23.6 million for the quarter ended December 31, 2019, a decrease of $0.9 million as compared to the immediate prior quarter ended September 30, 2019.

Net loss was $41.3 million or $1.36 per share for the third quarter of 2019 compared with a net loss of $26.4 million or $0.87 per share for the same period in the prior year.

At December 31, 2019, total cash and cash equivalents were $131.9 million. As you're aware, in December of last year, we entered into a $300 million low-interest, interest-only 5-year term loan facility with DSP with no repayments due until the end of the term.

At closing, we drew down $87.5 million and still have $212.5 million available to us under this facility, eliminating the need for any short-term equity financing.

In addition, DSP also expects to continue to support Urovant through profitability. Looking ahead, for the fourth quarter of fiscal 2019, ending on March 31, we expect our total operating expenses to be in the range of $41 million to $43 million, which includes a $10 million milestone payment that will become due upon acceptance of our NDA submission by the FDA.

Cash used in the quarter ending March 31, 2020, will include the $48.2 million that we used to repay the Hercules Capital loan and the $10 million milestone payment due upon acceptance of our NDA. We expect to end fiscal 2019 with a cash balance of $44 million to $46 million.

Turning now to fiscal 2020. We expect fiscal 2020 to be an exciting year for us as we prepare for the launch of vibegron during the first 3 quarters and then launch vibegron if approved by the FDA in the fourth quarter.

For fiscal 2020, which begins April 1, we expect our quarterly operating expenses to be higher than the first 3 quarters -- to be higher in the first 3 quarters as compared to approximately $30 million of quarterly operating expenses, excluding stock-based compensation for fiscal 2019.

Operating expenses will increase further in the fourth quarter of fiscal 2020 as we bring our sales force onboard and launch vibegron if approved by the FDA.

With that financial update, let me turn the call back over to Keith.

--------------------------------------------------------------------------------

Keith A. Katkin, Urovant Sciences Ltd. - CEO & Director [6]

--------------------------------------------------------------------------------

Thanks, Ajay.

I'd like to reiterate our excitement for what we have accomplished during this past quarter, especially, the progress we have made with the submission of our New Drug Application for vibegron and the initiation of our Phase IIa study for URO-902.

Our new relationship with DSP provides us with a stronger and more stable financial profile, while also providing access to a global platform, enhanced commercialization resources and other significant advantages as we prepare for the launch of vibegron.

We remain very excited about the market opportunity for vibegron in OAB. Our belief in the differentiation of vibegron is further supported by what we believe has been a very strong launch in Japan.

While the market dynamics in Japan are very different than the United States, current data suggests that vibegron was able to take meaningful share from mirabegron in a short period of time.

In closing, the third fiscal quarter of 2019 was transformational for our company, marked by key milestones across all aspects of our business.

We look forward to several upcoming milestones that will continue to drive towards the goal of developing Urovant into a leading specialty urology company. These upcoming milestones include: the FDA acceptance of our New Drug Application for vibegron in OAB, which we anticipate by the end of Q1 2020; our 2 upcoming presentations in May at AUA in Washington, D.C., that includes data from our recently completed post hoc study, demonstrating the statistically significant benefit of vibegron over tolterodine at week 52; Phase IIa top line safety data for IBS-associated pain in the second half of 2020; the continued and the patient of cohort 1 enrollment in Phase IIa of our novel injectable gene therapy for OAB URO-902, followed by the initiation of cohort 2 enrollment later this year; and the ongoing evaluation of Part 2 of the Phase III COURAGE trial, which will assess both efficacy and safety of vibegron in men with OAB and BPH.

With that, I'll now open the call to questions. Operator, can you open the line?

================================================================================

Questions and Answers

--------------------------------------------------------------------------------

Operator [1]

--------------------------------------------------------------------------------

(Operator Instructions)

Your first question comes from the line of Mr. Eric Joseph from JP Morgan.

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Eric William Joseph, JP Morgan Chase & Co, Research Division - VP & Senior Analyst [2]

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You kind of anticipated one of them here. I'm just curious to get a better of a sense of what you're seeing from Kyorin and their launch of Biova in Japan? And -- understanding there's going to be differences in dosing in the label here in the U.S., but where are they seeing patient demand come from? And what's the dynamic been with mirabegron there?

--------------------------------------------------------------------------------

Keith A. Katkin, Urovant Sciences Ltd. - CEO & Director [3]

--------------------------------------------------------------------------------

Yes. Thanks for the question, Eric.

We don't have a ton of information from Japan, particularly, we don't have insight into exactly what patients are going after with their campaign. What we do have is that we've got 2 sources of information: we've got essentially the Japanese IQVIA data; and also and maybe more importantly, we've got the sales numbers that have been reported by Kyorin as they're a publicly traded company.

Both the IQVIA-like data in Japan and also the sales numbers that we're seeing from Kyorin suggest they're taking a really nice percent share. We don't want to go into the specifics, but even if we got that share within the first 12 months of our launch, we would be extraordinarily happy with the uptake of our launch as we think everybody else would be as well.

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Edited Transcript of UROV.OQ earnings conference call or presentation 13-Feb-20 9:30pm GMT - Yahoo Finance

Recommendation and review posted by Bethany Smith

Novel therapies developed in the past several decades may have improved long-term survival for patients with multiple myeloma (MM) undergoing autologous stem cell transplantation (ASCT), with more patients in the past several years having what approaches a normal life expectancy, according to research published in Blood Advances. Outcomes differ, however, depending on patient age, disease risk group, and cytogenetic characteristics.

Previously published data suggest that patients with MM had the best survival outcomes in the period between 2002 and 2004, with a 5-year relative survival of 34.7%. However, inevitable disease relapses have been found to occur at increasingly short intervals in this patient population.

ASCT is a further mainstay of MM management, and there are limited data regarding outcomes among patients who undergo the total therapy approach, which involves induction therapy, ASCT, maintenance, and novel therapies as they become available for use. For this study, researchers evaluated outcomes among patients with MM who underwent total therapy and for whom long-term data were available.

Overall, 4329 patients were included in the analysis, with a median age of 58.9 years (range, 17.4-84.9) and 72.1% of patients being younger than 65 years; 2646 patients (61.1%) were male, 3723 (86%) were Caucasian, and 1861 (43%) were enrolled in a total therapy clinical trial. Patients were also categorized by the year in which they underwent ASCT: 1997 or earlier (661 patients), 1998 to 2003 (1002 patients), 2004 to 2008 (1294 patients), 2009 to 2013 (837 patients), and 2014 or later (535 patients).

The overall median follow-up was 10.5 years (range, 0.01-26.4), with patients who received ASCT in 1997 or earlier having the longest median follow-up (21.5 years) and those who received ASCT in 2014 or later having the shortest (2.5 years).

Overall survival improved over the evaluated period, with patients who received ASCT in 2014 or later having a hazard ratio for mortality of 0.35 compared with patients in the 1997 or earlier group (early mortality, 32.3% vs 36.4%). Other independent predictors of early mortality across the patient sample included age of 65 years or greater (37.2% vs 27.6% in patients younger than 65 years), high Gene Expressing Profile 70 risk status (61.4% vs 17.5% for patients with low risk), and presence of a chromosomal abnormality (41.7% vs 22.8% for patients with no abnormalities).

Being enrolled in a total therapy clinical trial was also associated with better early mortality (20.9% vs 37.9% for patients not on a total therapy protocol).

Going forward, it is clear that continued efforts to control and eradicate residual disease will be important strategies to increase the likelihood of achieving MM cure, the researchers wrote.

Reference

1. Nishimura KK, Barlogie B, van Rhee F, et al. Long-term outcomes after autologous stem cell transplantation for multiple myeloma. Blood Adv. 2020;4:422-431. doi:10.1182/bloodadvances.2019000524

Read more here:
Total Therapy Approach May Improve Transplant Outcomes in Patients With Myeloma - Hematology Advisor

Recommendation and review posted by Bethany Smith

The latest research analysis titledGlobalGene TherapyMarketgives a detailed assessment of the market where each factor, components, segments, and other sections of the market are comprehensively described. The report forecasts theGene Therapymarket to portray prominent growth during the forthcoming years from 2019 to2025. The report delivers geological study into several regions with market growth, production, consumption, and revenue. The research report focuses on critical data that makes it a very important tool for research, analysts, experts, and managers. It examines data and estimates on the market structure, dynamics, and trends.

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Global Gene Therapy Market 2019 Research Strategies and Forecasts till 2025 - Galus Australis

Recommendation and review posted by Bethany Smith

This research study on Gene Therapy for Rare Disease market reports offers the comparative assessment of Gene Therapy for Rare Disease market and consist of Historical data, Significance, statistical data, size & share, Market Price & Demand, Business overview, Market Analysis By Product and Market Trends by Key Players. This Gene Therapy for Rare Disease Market is Segmented in two type on the basis of type of materials and end-users. It has global market covered in all the regions, ranging to that fundamental market, key trends and segmentation analysis are coated throughout Gene Therapy for Rare Disease market report.

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Recommendation and review posted by Bethany Smith

Gene Therapy for Inherited Genetic Disorders Market report is designed by detailed investigation procedure to collect all the necessary data. This report contains the brief profile of leading players in the industry along with their future plans and current developments. Further, report considers the revenue generated from the market analysis and opportunity analysis to estimate the market size. The report initiates with the basic market outlook and structure along with forecast of the various segments and sub-segments.

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The key players covered in this study, BioMarin Pharmaceutical Inc., bluebird bio Inc., Novartis AG, Orchard Therapeutics Plc, Spark Therapeutics Inc.,

No of Pages: 95

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Market segment by Type, the product can be split intoEye DisordersHematological DisordersCentral Nervous System DisordersMuscular DisordersOthersMarket segment by Application, split intoHospitalClinicResearch InstituteOthers

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Table of Contents

1 Study Coverage

2 Executive Summary

3 Breakdown Data by Manufacturers

4 Breakdown Data by Type

4.1 Global Gene Therapy for Inherited Genetic Disorders Sales by Type

4.2 Global Gene Therapy for Inherited Genetic Disorders Revenue by Type

4.3 Gene Therapy for Inherited Genetic Disorders Price by Type

5 Breakdown Data by Application

5.1 Overview

5.2 Global Gene Therapy for Inherited Genetic Disorders Breakdown Data by Application

6 North America

7 Europe

8 Asia Pacific

9 Central & South America

10 Middle East and Africa

11 Company Profiles

12 Future Forecast

13 Market Opportunities, Challenges, Risks and Influences Factors Analysis

14 Value Chain and Sales Channels Analysis

15 Research Findings and Conclusion

16 Appendix

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Gene Therapy for Inherited Genetic Disorders Market by Key Players, Deployment Type, Applications, Vertical, and Region-Global 2026 Forecast -...

Recommendation and review posted by Bethany Smith

Idaho State Capitol

Madison Hardy

Madison Hardy is a Boise High School alum who grew up surrounded by the citys political buzz and vibrant culture. With a driving passion for the arts, history, and community relations Madison has enjoyed watching Idaho expand over the last 12 years.

Madison is covering the 2020 state legislative session in Boise, Idaho, as an intern for the University of Idaho School of Journalism & Mass Media, the McClure Center for Public Policy Research. A senior at the University of Idaho, Madison is graduating in May 2020 with a bachelors degree in Broadcast Journalism & Digital Media and a History minor.

You can follow her twitter account at @madisonhardy05.

BOISE -- Gender equality is a contentious issue in the United States, even in factors as specific as school sport programs. In Wednesdays House Education Committee meeting, Feb. 12, Republican Idaho Falls Representative Barbara Ehardt says there is a new challenge for sportswomen: transgender athletes.

You see opportunities continually taken away from girls and women, and you see girls and women being forced into an arena where it is not fair and they cannot compete in, said Rep. Ehardt. In some instances, they are literally in danger.

Ehardt says the proposed legislation will ensure fair athletic opportunities for girls and women by preventing male-to-female transgender students from competing in female sporting events.

This bill will center on opportunities for girls and women because those opportunities for boys and men, they [male-to-female transgender students] still have them, they just need to do it on that side, said Ehardt.

Language in the bill bases a students participation in school sports teams on their hormones, internal and external organs, and chromosome makeup. Originating from fairness for girls and women, it would encompass all transgender athletes to be defined by their birth given sex. The bill, also sponsored by Senator Mary Souza (R- Coeur dAlene), would define a students gender in three ways; physiologically, chromosomally and hormonally.

Some democratic members of the committee, like Rep. Steve Berch (D - Boise) and Rep. John McCrostie (D - Garden City), openly did not support introducing the bill.

I just think that this is an issue that is so far down the priority list that we have in this state facing education, said Rep. Berch. I just don't think this is where we should be spending our time.

Other members of the committee disagreed.

I hardly think equality in sports is seen as an unimportant issue for this committee to be undertaking, Rep. Gayann Demordaunt (R - Eagle) said.

The current policy in Idaho requires that a male-to-female transgender student athlete must complete one year of medically prescribed hormone treatment under a physicians care related to the gender transition before competing on the female team. However, they are permitted to participate on the male team without limitations.

I think the most important thing to share is that this RS [bill] would actually promote the notion that trans-girls are not real girls and that trans-women are not real women, said Rep. McCrostie. I find it disappointing.

Ehardt says the bill does not intend to discriminate LGBTQ students, and she has said she personally supports transgender persons.

Idaho is not alone in discussing transgender athletes in school programs. Recently legislators in approximately five states proposed legislation that prevents athletes from competing in categories different from their biological sex. The states with introduced or prefiled legislation are New Hampshire, Washington, Georgia, Tennessee and Missouri, the Wall Street Journal reported.

The vote to introduce the bill passed, but was divided on party lines. Next stop will be a public hearing in an upcoming House Education Committee meeting.

Continue reading here:
New bill prohibits transgender students from participating in women's sports - Idaho County Free Press

Recommendation and review posted by Bethany Smith

TORRINGTON A pathologist testifying in murder trial Thursday outlined that a Guernsey man who died in a stabbing suffered a fatal wound to his heart.

Dr. Peter Schilke, a forensic crime pathologist with Western Pathologist Consultants in Scottsbluff, outlined his findings in the murder trial of Jamie Snyder. Snyder, 28, is charged with first-degree murder in the May 24, 2018, stabbing death of 32-year-old Wade Erschabek, of Guernsey.

Schilke performed the autopsy on Erschabeks body. Schilke had been contacted by Goshen County Coroner on May 24, 2018, to perform the autopsy following the stabbing, which occurred in Fort Laramie.

Schilke said Erschabek had been stabbed on the left side of the chest and he produced photos of the exam. He showed the jury a photo of the heart where a cut to the left ventricle was shown, which Schilke said was the cause of death.

The stab wound was in the mid-left chest and went through the left forth-fifth-sixth ribs and cut the pericardium and the left ventricle, also cutting the left lobe of the lung. Erschabek had about a liter of blood in his chest, he said.

Deputy Prosecuting Attorney Jeremiah Sandburg showed the black knife identified as the murder weapon to Schilke and asked if it was the kind of knife that could have caused that injury. Schilke said it could have. The knife wound was 5 -inches deep and 2 -inches in length. His final conclusion was that the stab wound to the heart resulted in homicide.

In the days prior to the stabbing, friends described Snyder as having been acting erratically and making threats.

Clayton Paules took the stand and said Snyder told him someone had broke into his house and stolen items. Snyder allegedly suspected Erschabek or another man. The other man had been in the hospital at the time of the reported burglary, according to his testimony.

Later, Clayton Paules said, Snyder showed up at his home, unannounced, walking and wearing a holster with a knife in it. Clayton Paules described Snyder as in a very distressed mood and pretty worked up about the burglary. Defense attorney Jonathan Foreman asked if Snyder was kind of acting wild, a characterization that Clayton Paules agreed with. He said he had friends over and they were kind of freaked out by him and scared. He said they all left.

Michael Paules, who testified that he had tried to be a father figure to Snyder, also described Snyder as being upset before the stabbing. According to Michael Paules, Snyder told him Erschabek needed to die.

He looked really different, like he was really holding a grudge, Michael Paules said. He didnt seem outraged or nothing like that. He had no guns or knives when I was with him. He was just holding a grudge. And, this was before any talk of the burglary.

When Michael Paules heard about the burglary and saw Snyder, he said the man had changed, wearing all back and acting all Goth.

Michael Paules said he tried to redirect the man, telling him, Why dont you think of your daughter or something else instead of killing someone? He said he did not take Snyders threats seriously.

During Wednesdays proceedings, defense attorney Jonathan Foreman, asked Eighth Judicial District Judge Patrick Korell for a summary judgment, asking the judge to dismiss the case or reduce charges to second-degree murder or manslaughter in the case against Snyder. Foreman argued that the state had not proven its case for first-degree murder.

After a break, Korell denied the motion, saying that the state had met the requirements for considering first-degree murder in this case. The trial then moved into the defense phase, with Snyders attorney calling Dr. Katherine Mahaffey, a physician at the Wyoming State Mental Hospital in Evanston, to the stand.

Mahaffey testified about Snyders mental health, saying he had Snyder with a personality disorder, antisocial behavior disorder. She had met with him twice previously before he spent 3 1/2 months in the hospital, from November 2019 to January 2020.

According to questioning from Foreman, another doctor had previously treated Snyder for mental health issues diagnosing him with paranoid schizophrenia in 2012 and treating him at Regional West Medical Center. Mahaffey testified about Snyders mental health history, saying the man had also been diagnosed with borderline psychotic with narcissistic social disorder, but the man also showed drug induced symptoms from ecstasy, methamphetamine and marijuana use. Foreman asked about Snyder experiencing symptoms of grandiose thinking, impulsivity and paranoia, with interpersonal conflict, which Mahaffey agreed with and noted as being under the same umbrella of mental illness. The man had also been experiencing a lot of stress, having lost a job and moving back to Fort Laramie to live with his mother.

According to the testimony, on the night of Snyders arrest, he had told his mother he saw tri-colored people with guns. Snyder had trouble keeping on a subject when talking or questioned, changing the subject.

Prior to his arrest, Sndyer told Mahaffey that he had been taking pre-workout drinks, full of caffeine; dhea, a hormone supplement; anabolic steroids and Vitamin B. He described himself as not sleeping well and having lost lots of weight, she testified.

Testimony in the case will resume on Thursday in Goshen County District Court.

We're always interested in hearing about news in our community. Let us know what's going on!

Read more:
Pathologist: Stabbing victim died of fatal wound to heart - Scottsbluff Star Herald

Recommendation and review posted by Bethany Smith

Its hard to ignore hunger, a powerful drive designed to get your attention and keep the body from starving.

Regular meals that fill the stomach and intestines should calm it down, but what if youre always ravenous for another bite of food, no matter how much you eat?

Hunger is complicated and can have many different triggers, said Dr. Monique Tello, a clinical instructor at Harvard Medical School, practicing physician and director of research and academic affairs for the healthy lifestyle program at Massachusetts General Hospital.

One is hormonal, so peoples hormones in particular ghrelin, a gut hormone can have a stimulating effect on the sensation of hunger and appetite, Tello, author of Healthy Habits for Your Heart, told TODAY.

Other signals are more psychological, and these are very commonly the trigger for peoples hunger.

First, its important to rule out any medical issues. Anybody who is feeling very hungry all of the time and isnt able to gain weight or is losing weight should see a doctor, Tello said.

Conditions that could cause constant or excessive hunger, also called polyphagia, include:

Hyperthyroidism: When the thyroid is overactive, a persons body and metabolism are all revved up, Tello noted. Besides being hungry, patients feel jittery, shaky and their heart may be racing.

Diabetes: People with type 1 diabetes lose the ability to make insulin so their body cant process sugar. Theyre usually telling me: Im eating and eating, Im losing weight and I feel terrible, Tello said. Hunger can also be a symptom of type 2 diabetes, where the body is resistant to insulin.

Damage to the hypothalamus: This part of the brain helps regulate feelings of appetite and satiety. If its damaged because of a tumor or head trauma, it can cause uncontrollable hunger and hypothalamic obesity.

Drugs like prednisone a corticosteroid steroid commonly used as an anti-inflammatory or an immunosuppressant medication can also increase hunger, said Beth Kitchin, an assistant professor of nutrition at the University of Alabama at Birmingham. So can some hormone therapies for women going through menopause.

Trending stories,celebrity news and all the best of TODAY.

She always asks people who complain theyre constantly hungry whether they have started taking any new medicines or changed their doses.

If there isnt an underlying medical issue, the problem could be in the head.

Its reasonable to be hungry every three to five hours given how the human digestive system works, Kitchin said. But ever-present food marketing on TV and the constant stream of food porn on social media can trigger people to eat often and a lot, both experts noted.

There are also deeply ingrained cultural triggers, like the idea of eating three meals a day plus snacks, Tello said. She hates the myth of breakfast or the notion people have to eat as soon as they wake up.

I tell patients, The more you eat, the more you want to eat, Tello said.

The more people eat, the larger the stomach gets. The stomach can stretch to accommodate large amounts of food its a distensible organ. Then if its empty, it signals hunger. Well, if youve got a huge stomach from eating so much so often, the minute your stomach is empty, its signaling you to eat and youre going to eat more.

A persons state of mind can play a role, too. Stress can increase levels of ghrelin, research has shown, and being sleep deprived is associated with higher levels of the hunger hormone.

Boredom, anxiety and depression can also send people looking into the refrigerator when theyre not truly hungry.

Sometimes the best answer to the question Why am I always hungry? is the simplest one: Youre eating too little, exercising too much, or both.

Kitchin often sees it at the beginning of the year when patients go too far with their New Years resolutions.

When people tell me Im so hungry, I look at their food diaries and I can see why theyre hungry. Theyre just not eating enough sometimes, she said.

Dont make yourself hungrier than you need to be: Limit your exposure to TV and social media. Try to watch your favorite shows without being exposed to advertising, Tello said. She gets her groceries delivered so she can avoid being bombarded by food marketing in the grocery store.

Get honest: Ask yourself, "Am I really hungry? Or am I bored?" Remove yourself from any food temptations if it's the latter. Get help to deal with anxiety or depression rather than self-medicating with food.

Consider intermittent fasting: It can reconnect you with true, biological hunger; make it easier to recognize feeling full; provide daily structure and break the habit of snacking, experts say.

Going without food for a while can help the stomach to shrink, and help people to eat less and experience hunger less, Tello noted.

I have patients who eat one meal a day and they are fine. I have an aunt who never eats breakfast, she doesnt feel hungry until lunch time and shes at a really healthy weight, Kitchin added.

Feel fuller by adjusting the quality of your diet: Avoid processed carbohydrates and sugars found in foods like white bread, baked goods and cereal. Aim for a satiating diet higher in fiber, protein and healthy fats, Tello advised. Dip carrots into some peanut butter, enjoy a hard-boiled egg or munch on an apple. Such choices will keep you more satisfied, longer.

Foods that naturally contain a lot of water cucumbers, tomatoes, strawberries, grapes and so on are more satiating than other options even though they have fewer calories, said Dr. Michael Greger, author of How Not to Diet: The Groundbreaking Science of Healthy, Permanent Weight Loss.

These higher-volume options take longer to eat, which seems to signal the brain that youre filling up.

Watch your alcohol intake: Alcohol lowers a person's inhibitions and self-discipline, which can make you eat more.

Read more:
Why do I always feel hungry? Why you eat even when you're full and how to stop - TODAY

Recommendation and review posted by Bethany Smith

February is Heart Awareness Month, which now makes the perfect time to educate yourself about arguably one of the most important parts of the body, likely only competing with the brain for top rank.

Without knowledge about our blood-circulating machine, we put ourselves at risk for mistreating our bodies and putting our health at risk and potentially leading ourselves to the possibility of a shorter life span and possibly even to expensive medical procedures down the line.

Heart disease and other cardiovascular issues do not arise in only older adults and can occur in any individual due to a variety of health factors. Our heart health is affected throughout our lives, and without proper treatment and care for the cardiovascular system, it is more likely that the onset of potential health struggles may occur faster than one might expect, and they are hard to reverse once the snowball effect of cardiovascular decline is set in motion.

High rates of obesity and high blood pressure are climbing in the younger generations of America and other countries, and this puts them at risk for heart problems at earlier ages. Some serious threats to heart health include tobacco use, mainly smoking tobacco, high blood pressure and high blood cholesterol levels.

Obesity puts stress on the heart and can cause other health issues that may add strain to the cardiovascular system, such as diabetes and physical inactivity, which both increase the likelihood of a more rapid decline in heart health.

Unhealthy eating patterns can lead to other unhealthy habits, which can start the aforementioned snowball rolling and exponentially increase your risk of heart issues at any age.

Some ways to take the best care of your heart include getting regular exercise, which can be helped by finding a set schedule you stick to keep yourself disciplined and well-motivated. If regular exercise has proven itself to be a difficult goal for you, finding a workout buddy or a certain activity you enjoy will make working out less of a chore and more of an enjoyable experience for you as well as your workout companion.

Hours of cardio are not required to keep your heart healthy, but some moderate exercise for at least 150 minutes per week will keep your heart healthy and happy. It may seem daunting to face 150 minutes head-on, but splitting that time into 30-minute blocks makes regular physical activity seem much more manageable and enjoyable. Regular exercise has also been proven to increase serotonin levels, which is known as the happy hormone.

Getting regular check-ups with a physician, usually necessary only once a year, is the perfect way to stay updated on the condition of your cardiovascular system. Remaining aware of the status of your heart health is vital in order to keep an eye on any potential conditions you may need to address and manage with your health care professional.

The food you eat plays a large role in the condition of your cardiovascular system as well. Keeping a diet full of whole grains, veggies and healthy fats while limiting your intake of processed sugars and saturated fat is ideal.

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Sustaining the art of the heart during Heart Awareness Month - Indiana University The Penn Online

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San Francisco, California (UroToday.com) Dr. Piet Ost provided the radiation oncology perspective for the Best of the Journals in Prostate Cancer Session at the 2020 American Society of Clinical Oncology Genitourinary Cancers Symposium (ASCO GU), discussing several important trials that published results over the past year.

The first trial Dr. Ost discussed was the trial of ultra-hypofractionated versus conventionally fractionated radiotherapy for prostate cancer: 5-year outcomes of the HYPO-RT-prostate cancer randomized, non-inferiority, Phase III trial.1 The Scandinavian HYPO-RT-PC randomized controlled Phase III trial was initially presented at ESTRO 2018 and was subsequently published in Lancet Oncology. This trial randomized men with intermediate and high-risk prostate cancer to either conventional fractionating (n = 602; 78.0 Gy in 39 fractions, 5 days per week for 8 weeks) or ultrahypofractionated (n=598; 42.7 Gy in seven fractions, 3 days per week for 2.5 weeks).

The primary endpoint was time to biochemical or clinical failure. The estimated failure-free survival at five years was 84% (95% confidence interval [CI] 80-87) in both treatment groups, with an adjusted hazard ration (HR) of 1.002 (95% CI 0.758-1.325; log-rank p=0.99). There was weak evidence of an increased frequency of acute physician-reported RTOG grade 2 or worse urinary toxicity in the ultra-hypofractionation group at end ofradiotherapy(158 [28%] of 569 patients vs 132 [23%] of 578 patients; p=0.057). Based on these results, there has been support for the use of ultra-hypofractionated radiotherapy for prostate cancer.

The second trial Dr. Ost presented was the intensity-modulated fractionated radiotherapy versus stereotactic body radiotherapy for prostate cancer (PACE-B) trial assessing acute toxicity from an international, randomized, open-label, Phase III, non-inferiority trial.2 Patients were either low-risk or intermediate-risk prostate adenocarcinoma (Gleason 4+3 excluded), and scheduled to receive radiotherapy were recruited from 37 centers. Participants were randomly allocated (1:1), stratified by center and risk group, to conventionally fractionated or moderately hypofractionated radiotherapy (78 Gy in 39 fractions over 7.8 weeks or 62 Gy in 20 fractions over four weeks, respectively) or stereotactic body radiotherapy (36.25 Gy in five fractions over 1-2 weeks).

The primary endpoint of PACE-B is freedom from biochemical or clinical failure. There were 874 men to conventionally fractionated or moderately hypofractionated radiotherapy (n=441) or stereotactic body radiotherapy (n=433). Worst acute RTOG gastrointestinal toxic effect proportions were as follows: grade 2 or more severe toxic events in 53 (12%) of 432 patients in the conventionally fractionated or moderately hypofractionated radiotherapy group versus 43 (10%) of 415 patients in the stereotactic body radiotherapy group (difference -19 percentage points, 95% CI -6.2 to 2.4; p=0.38). Worst acute RTOG genitourinary toxicity proportions were as follows: grade 2 or worse toxicity in 118 (27%) of 432 patients in the conventionally fractionated or moderately hypofractionated radiotherapy group versus 96 (23%) of 415 patients in the stereotactic body radiotherapy group (difference -42 percentage points, 95% CI -10.0 to 1.7; p=0.16).

The third trial Dr. Ost discussed was the short-term androgen deprivation therapy (ADT) continued with radiotherapy as salvage treatment after radical prostatectomy for prostate cancer (GETUC-AFU 16).3 This study included patients with stage pT2, T3, or T4a (bladder neck involvement only) and pN0 or pNx according to the TNM staging system, whose prostate-specific antigen (PSA) concentration increased from 0.1 ng/mL to between 0.2 ng/mL and 2.0 ng/mL after radical prostatectomy, without evidence of clinical disease. Patients were assigned (1:1) to short-term androgen suppression (subcutaneous injection of 10.8 mg goserelin on the first day of irradiation and 3 months later) plus radiotherapy (3D conformal radiotherapy or intensity-modulated radiotherapy of 66 Gy in 33 fractions, five days a week for seven weeks) or radiotherapy alone.

Randomization was stratified according to investigational site, radiotherapy modality, and prognosis. The primary endpoint was progression-free survival (PFS) in the intention-to-treat population. There were 743 patients randomly assigned, 374 to radiotherapy alone and 369 to radiotherapy plus goserelin. At the time of data cutoff, the median follow-up was 112 months (IQR 102-123). The 120-month PFS was 64% (95% CI 58-69) for patients treated with radiotherapy plus goserelin and 49% (43-54) for patients treated with radiotherapy alone (HR 0.54, 0.43-0.68; stratified log-rank test p<00001).

The final study Dr. Ost discussed was the STOpCaP systematic review recently published in European Urology.4 This systematic review included trials that randomized men to prostate radiotherapy and ADT or ADT only. The review identified one ongoing (PEACE1) and two completed (HORRAD and STAMPEDE) eligible trials. Pooled results of the latter (2126 men; 90% of those eligible) showed no overall improvement in survival (HR 0.92, 95% CI 0.81-1.04, p = 0.195) or PFS (HR 0.94, 95% CI 0.84-1.05, p=0.238) with prostate radiotherapy. There was an overall improvement in biochemical progression (HR 0.74, 95% CI 0.67-0.82, p < 0.0001) and failure-free survival (FFS) (HR 0.76, 95% CI 0.69-0.84, p < 0.0001), equivalent to 10% benefit at three years. The effect of prostate radiotherapy varied by metastatic burden-a pattern consistent across trials and outcome measures, including survival (<5, 5; interaction HR 1.47, 95% CI 1.11-1.94, p=0.007). In general, there was a 7% improvement in three-year survival in men with fewer than five bone metastases.

Dr. Ost concluded with several take-away points:

Written by:Zachary Klaassen, MD, MSc, Assistant Professor of Urology, Georgia Cancer Center, Augusta University/Medical College of Georgia, Twitter: @zklaassen_mdat the 2020 Genitourinary Cancers Symposium, ASCO GU#GU20, February 13-15, 2020, San Francisco, California

References:

1. Widmark, Anders, Adalsteinn Gunnlaugsson, Lars Beckman, Camilla Thellenberg-Karlsson, Morten Hoyer, Magnus Lagerlund, Jon Kindblom et al. "Ultra-hypofractionated versus conventionally fractionated radiotherapy for prostate cancer: 5-year outcomes of the HYPO-RT-PC randomised, non-inferiority, phase 3 trial."The Lancet394, no. 10196 (2019): 385-395.

2. Brand, Douglas H., Alison C. Tree, Peter Ostler, Hans van der Voet, Andrew Loblaw, William Chu, Daniel Ford et al. "Intensity-modulated fractionated radiotherapy versus stereotactic body radiotherapy for prostate cancer (PACE-B): acute toxicity findings from an international, randomised, open-label, phase 3, non-inferiority trial."The Lancet Oncology20, no. 11 (2019): 1531-1543.

3. Carrie, Christian, Nicolas Magn, Patricia Burban-Provost, Paul Sargos, Igor Latorzeff, Jean-Lon Lagrange, Stphane Supiot et al. "Short-term androgen deprivation therapy combined with radiotherapy as salvage treatment after radical prostatectomy for prostate cancer (GETUG-AFU 16): a 112-month follow-up of a phase 3, randomised trial."The Lancet Oncology20, no. 12 (2019): 1740-1749.

4. Burdett, Sarah, Liselotte M. Boeve, Fiona C. Ingleby, David J. Fisher, Larysa H. Rydzewska, Claire L. Vale, George van Andel et al. "Prostate radiotherapy for metastatic hormone-sensitive prostate cancer: a STOPCAP systematic review and meta-analysis."European urology76, no. 1 (2019): 115-124.

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ASCO GU 2020: Best of the Journals in Prostate Cancer - Radiation Oncology Perspective - UroToday

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FOUNTAIN When a child protection caseworker told Crystal Bryant they were taking her 5-month-old boy, the young mother dropped to her knees on the hospital floor.

Take me, she pleaded. For my son to stay home, take me, I dont care.

She begged God and anyone listening, but they still took him, Bryant recalled, shaking her head and wiping tears as she recounted the worst moment of her life. Child welfare and police officers made Bryant and her husband, Jarvis Bryant, leave the Colorado Springs hospital room before a foster mother arrived to take their baby away.

Their lives were shattered.

The Bryants, who moved to Fountain when Jarvis was assigned to Fort Carson, were charged with felony child abuse, accused of breaking multiple bones in their only childs body. Crystals recently earned nursing certificate was revoked. Jarvis, a U.S. Army specialist, lost his security clearance, stripping him of his job in aircraft and vehicle supply and relegating him to paper shuffling.

Worst of all, baby Jace was gone. Their apartment seemed empty. They felt powerless, more than lost like death, Crystal said.

The Bryants son was one of 4,772 Colorado children removed from their homes by child welfare authorities and living in foster care last year. But their story isnt the typical tale of a child rescued from abuse or neglect. Its the opposite. The Bryants nightmare lasted 164 days, and its end arrived more like a ripped-from-the-headlines television drama than real life.

Baby Jace had been sick his whole life. He had severe acid reflux and a swallowing condition called dysphagia that caused milk to go down his windpipe instead of his esophagus, making him choke.

When he was 3 weeks old, he spent 31 days in the hospital and had a feeding tube inserted into his nose and down to his stomach to help him get enough nutrients to survive. At almost 4 months old, Jace had surgery on his esophagus and to place a gastrostomy tube in his abdomen, so his parents could send formula straight to his stomach.

Then, just before Thanksgiving 2018, Jace was diagnosed with a viral infection that was causing fever and diarrhea. While Crystal showered and did her hair upstairs in their apartment, Jarvis was giving Jace his second bath of the day. Jarvis laid the baby stomach-down along his forearm as he bent over the bathtub, pouring soapy water over Jaces back. The baby slipped sideways off Jarvis arm, his knee hitting the bottom of the tub.

Jace cried, and Jarvis picked him up to console him, he said. Jarvis said he was leaning into the tub when the baby fell into a small amount of water, enough to make a toy float. Jarvis didnt mention it to his wife.

Later that Sunday afternoon, though, Crystal noticed Jace was favoring his leg, that he held it up as he sat in his vibrating saucer-chair and flinched when she picked him up. She was bringing him back to the hospital anyway because the fever and diarrhea had not subsided, and while she was there Crystal asked the doctor if they could X-ray Jaces leg.

The baby had a fractured femur.

Crystal called Jarvis at home, and he told her then how Jace had slipped from his arm and hit his knee. As Jarvis drove to the hospital, Crystal told the doctor that her husband had dropped their son in the bathtub.

Instantly, they were suspects.

Medical staff at Memorial Hospital Central ordered a full-body workup. What they found was devastating: Not only did Jace have a broken leg, he had 10 fractured ribs and two fractured wrists. The leg fracture was fresh, but the rest were in various stages of healing.

The police came, interviewing Crystal, 24, and Jarvis, 27, in separate rooms. A caseworker arrived from the El Paso County child welfare division, and a pediatrician with training in child abuse examined Jace. A detective did voice stress analysis tests by recording and analyzing their voices, deciding both parents were deceptive Jarvis about knowing who caused Jaces injuries and Crystal about whether she hurt her son.

Jaces medical records as well as the child abuse charging documents, both reviewed by The Colorado Sun, do not mention unexplained bruises or burns red flags of child abuse. They do note marks on his chest, back and cheek, some of them crescent-shaped scratches. The Bryants told authorities one mark was a scar from his g-tube surgery and the others were likely from his parents or medical staff accidentally scratching him with fingernails or a ring while picking him up.

Because of Jaces feeding problems, nurses, physical therapists and occupational therapists all required by law to report any suspicion of child abuse had been to the Bryants home twice each week since he was 2 months old. They had stripped Jace naked weekly and weighed him on a portable scale placed on the table. Not one made any report of abuse or neglect.

The baby had been to emergency rooms multiple times, often to replace the tube down his throat when his arms flailed and pulled out the tube, common for babies. Each time, medical staff took an X-ray to make sure it was in the right place. As a first-time mom and self-described hovering parent, Crystal took Jace to the pediatrician at the first hint of illness.

Still, three days after she brought Jace to the hospital and asked for the leg X-ray, El Paso County authorities told the Bryants he wasnt going home with them their home, authorities said, wasnt safe for their son.

The Bryants returned to their apartment without their baby and gathered up everything they thought he might need at his foster home toys, clothes, his medicines for acid reflux, formula, shampoo, laundry detergent, Vaseline. They had no family nearby; Crystal is from Chicago, and Jarvis is from Louisiana.

The following morning, a distraught Crystal dialed the child welfare division. Five times. I am concerned. I am pissed off. Im angry, sad, frustrated, she said.

Its Thanksgiving. Can I see my son?

The answer was no. Crystal sat on the couch and cried.

Losing their son sent Crystal into heavy depression, so dark that she once opened the passenger door in their car as Jarvis drove 80 mph down the highway away from a supervised visit with Jace. I just wanted to jump out, she said. I couldnt believe I was still living on this Earth, without my son. It was hell on Earth.

There was no living for me.

Crystal stopped eating and drinking and grew so ill that Jarvis drove her to Evans Hospital on Fort Carson in early January, about a month and a half after Jace was taken away. But when Jarvis handed over his military ID at the checkpoint, the guard hesitated.

Do you know there is a warrant out for your arrest? the guard asked, Jarvis recalled. Did you not pay a ticket or something?

There was a warrant for Crystal, too. Felony child abuse charges, in addition to the civil case to determine whether to terminate their parental rights, had been filed against them. The class 3 felony is punishable by up to 16 years in prison.

The Bryants, neither of whom have any criminal history, were handcuffed and taken in separate military police cars and held on post for an hour and a half, when city law officers transported them to the El Paso County Jail. Mug shots. The whole orange jumpsuit. Everything, Jarvis said. They spent the night and were able to post bond the next morning.

While Crystal slipped further into depression, Jarvis was the fire.

He contacted the ACLU and the NAACP to see if they could help with legal defense, though Jarvis and Crystal were each assigned two public attorneys for the civil and criminal cases. He spent hours reading medical records and court cases. This is literally our lives, our sons life, our livelihood, our well-being, our mental state, pretty much everything that we could ever think of is at stake, he said. Im not going to sit here and let this happen. Its one thing if you know you did something wrong. But if you know you didnt do anything wrong, why are you going to go down innocent?

Jarvis went to every hospital and doctors office that had seen Jace, beginning with his May 22, 2018, birth at Evans Hospital. He collected 1,200 pages of medical records, along with discs of all of Jaces X-rays, including those taken before and after his surgery and tube placements.

They hardly slept. They fought, wondering if losing Jace would break them. They went to supervised visits at the Family Visitation Center, and signed up for individual therapy and parenting classes, trying to anticipate what the county would ask of them and speed up the process to getting their son back.

One sleepless night in March, Jarvis stumbled on a YouTube video of TV journalist Katie Couric interviewing two families in 2013 who were wrongly accused of child abuse after their children were found with broken bones. A Texas father was arrested after his 1-month-old daughter was found with nine bone fractures. Another father was accused of abusing his twin infants after they both had leg fractures. Both were eventually cleared because the babies all had a brittle bone disorder.

The video led Jarvis to a website called Fractured Families, a network for parents of children with unexplained fractures. And he fired off multiple emails titled Can you please help my family?

Through the network, Jarvis found contact information for more than a dozen physicians and radiologists who were experts in bone fractures and emailed all of them. We need help from a medical professional urgently to try and fight this matter properly and get our baby back and prove our innocence, he wrote. It just doesnt make any sense how a baby like ours has such a lengthy medical history and no one has done their due diligence to properly rule out everything before they automatically say child abuse. Please help us.

Two doctors agreed to help, for free.

Dr. Susan Gootnick, a radiologist in California who began studying alleged child abuse cases about seven years ago, looked at Jaces X-rays and noticed immediately that his bones appeared washed out because they werent getting enough calcium. It made sense, given Jace had not been able to eat by mouth for the first several months of his life. His scans were consistent with metabolic bone disease and rickets, she wrote in a report that Jarvis gave to his attorney.

Obviously, the baby wasnt getting the appropriate nutrients that he needed to grow, Gootnick said in an interview with The Sun. These bones break under stresses that a normal bone would not break under.

Whats more, Gootnick said, the age of the fractures made it likely that Jaces ribs and wrists were broken by medical staff during one of the many times they reinserted his nasal tube unsedated. An X-ray taken in September shows the rib fractures and the tube that went from Jaces nose to his stomach.

Its unpleasant, as you can imagine, sticking a tube down their nose, Gootnick said. You have to increase the strength of how you hold the baby still.

On one of the X-rays of Jaces wrist, Gootnick noted, an adult thumb bone is visible the technician holding down his arm for the scan.

Another doctor, Dr. John Galaznik, a retired pediatrician in Alabama who has testified for the defense in numerous child abuse trials, zeroed in on two acid-reflux medications Jace had taken since he was 3 weeks old Omeprazole and Ranitidine. They cut down on stomach acid, which is essential for absorbing calcium. Galaznik said it was likely that Jaces muscles so badly needed calcium that they were leaching it from his bones.

He cited the babys elevated levels of parathyroid hormone, which the brain secretes at a higher level when the bodys calcium requirement is not met, Galaznik said.

The doctor also noted that the fractured leg bone was consistent with a compressing force such as the bathtub fall Jarvis described. The fracture suggested no yanking, pulling, twisting forces as one might predict an abusing caregiver to inflict, he wrote.

Galaznik, who for 37 years was a physician in the University of Alabama system, said in an interview that the study of bone fractures in babies has evolved rapidly over the last 10 years, so much so that emergency department physicians who were trained 10 or 20 years ago are often not up to date.

In 2009, the American Academy of Pediatrics doubled the recommended vitamin D intake for babies, noting that vitamin D deficiency could lead to decreased calcium absorption and bone fractures. And in 2014, the academys Committee of Child Abuse and Neglect published a paper calling attention to the difference between fractures caused by abuse and those resulting from rickets or brittle bone disease, called osteogenesis imperfecta.

Galaznik is contacted by dozens of families each year and takes on some of the cases pro bono. He and Gootnick said they have noticed a societal bias against parents who are low-income and without higher education. If you come in and you are well-dressed and well-known and have no background problems, it may give the accusers more pause, Galaznik said. Just the allegation can destroy a career.

Gootnick said the cases she takes on lack sufficient evidence. There should be more investigation of whats going on with these kids in terms of their bone health, she said. I feel a moral obligation to help these people. If I can help them, I have to help them.

Jarvis printed out the doctors reports and gave them to his attorneys. And they waited.

Jace lived with the same foster family for 164 days. The Bryants counted all of them.

The baby, who was in the foster home from age 5 months to 10 months, seemed to know his parents less and less each week when they saw him for their two-hour supervised visit. One more of those and I feel like my son wouldnt even remember me, Crystal said.

They tried to move Crystals sister from Chicago so she could become Jaces foster mom, an arrangement the child welfare system calls a kinship placement. When she couldnt come, a couple Jarvis met at work offered to help. They became Jaces foster placement, and the Bryants were allowed to move into their house, under the condition that they were never alone with their son.

The arrangement lasted about two months, until the day last July when Jarvis and Crystal were to appear in civil court for their hearing on whether they could keep their son. Instead, the El Paso County child welfare division which in the meantime had deposed the divisions child abuse doctor in the wake of the pro-bono expert reports recommended they take their son home.

The Bryants, elated and relieved, took Jace home that night seven months after he was removed from their custody.

Three months later, in October, the criminal charges against them were dismissed. District Judge David Gilbert ordered the case sealed during the same hearing. It was like it happened and all of a sudden, it was wiped away, Jarvis said.

Crystal was in disbelief. I never would have thought in a million years that this would be our life, she said. I never thought this could happen to my son. But were still here. People would have thought we would have given up and turned on each other and Jace would stay in foster care. But I survived that. We survived that.

The Bryants, who are black, believe they were victims of a racial bias that exists in the child welfare system. A U.S. Department of Health and Human Services report showed that African American children are placed in foster care at almost twice the rate of white children, and they stay in foster care longer. Two studies in Texas found that even when African American families were assigned lower risk scores in child-abuse assessments, they were more likely than white families to have their children removed.

El Paso County child welfare officials declined to discuss the Bryants case, citing confidentiality laws. They said it is extremely rare that parents are accused of abuse and then exonerated, although they could not say how often it happens because the state child welfare data system does not keep track.

It is exceedingly, exceedingly rare, said Kristina Iodice, the departments public information officer. El Paso County investigated 6,633 child abuse and neglect allegations last year. Just more than 1,753 of those were founded. On any given day in 2019, about 360 children in the county were in foster placements.

Before a child is removed, the case is reviewed by a team that includes supervisors and medical professionals, said April Jenkins, intake manager for the countys Children, Youth and Families Services. A judge has to verbally approve a childs removal, no matter what time of day or night, and child protection workers have to present written evidence for the removal in court within 72 hours.

We are not making any decisions or assumptions or anything like that in a vacuum, Jenkins said.

On a recent afternoon, 20-month-old Jace squealed and giggled as he crawled into a play tent in the Bryants living room, where photos of Jace as a baby, the Bryants in military uniform shortly after they met at basic training in Missouri, and the couple on their wedding day hang on the walls. The Bryants have a new appreciation for days when the three of them are at home together, like when Jarvis made gumbo and they watched Louisiana State University win the national college football championship against Clemson.

Jace is eating by mouth now, after Jarvis and Crystal slowly introduced him to foods, starting with strawberries and other soft fruits. He is growing out of neonatal rickets and his bones are becoming stronger. And his g-tube is scheduled to come out in April. Jace doesnt seem to remember what he went through, but his parents think they will probably tell him, some day.

Crystal gave up on restoring her nursing certification the main reason she wanted it was to take care of Jaces g-tube. Jarvis is still waiting for the Army to restore his security clearance, and when it does the Bryants hope to leave Colorado for another Army post. The state where their baby was born has too many bad memories.

When they drive to Fort Carson, they avoid the guard station where they were arrested. When Jace was sick recently with a 104-degree fever, they hesitated before taking him to the hospital. Im sure as soon as you pull up his medical records, it says possible abuse, and everybody is looking at us now, Crystal said.

Wherever they live, the Bryants said they wont get over what they experienced during the past year. Theyre speaking out now to help other families who are falsely accused.

Its no hard feelings for us, but its anger, Jarvis said. This is what we actually had to live through. This is what our son had to live through. There should be due diligence before someones child is removed from their care. That is like, absurd, that you can take someones child and get a judge to sign off on it and you didnt even do your due diligence.

The Bryants said some of those involved in their case, including at the child welfare division, apologized privately for what they had been through. For Crystal, though, nothing can make up for lost time.

Im still grieving those days I didnt have my son with me, she said. We missed so many diaper changes, so many baths. I missed so much of his life.

This reporting is made possible by our members. You can directly support independent watchdog journalism in Colorado for as little as $5 a month. Start here: coloradosun.com/join

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Their baby was taken away and placed in foster care for 164 days. It turns out, he had a bone disorder. - The Colorado Sun

Recommendation and review posted by Bethany Smith

In the age of Instagram, self-care has actually ended up being associated with extravagances like massage therapies, facials, expensive items, store exercise courses as well as lush holidays.

That all audios fantastic if you have lots of non reusable revenue. But for the majority of us, investing severe cash money on self-care simply isnt reasonable.

The whole concept of self-care has really strayed from the original intent, and become a meme unto itself, claimed Kathleen Dahlen deVos, a therapist in SanFrancisco When I talk with my clients about self-care, rarely am I encouraging practices and habits that cost money. In fact, spending excessive money or funds we dont have In the name of self-care can actually be distressing, destructive and work against our mental and emotional wellbeing.

We asked professionals in the wellness area to share a few of the very best ways to practice self-care that are essentially totally free. Heres what they informed us:

Take a stroll around the block, being in the turf, trek a regional path or simply allow the sunlight radiate on your face for a couple of mins.

No matter where you live, you likely have access to an outside space, claimed Tiffany Lester, an integrative medication physician in SanFrancisco If its not in your neighborhood, think of a close space you can get to within 10 to 30 minutes. Getting outside and away from our devices calms our nervous system from the negative effects of everyday stressors.

When your home or workplace is a mess, it can take a toll on your psychological state, making you really feel a lot more stressed out, distressed as well as overloaded.

For some, a messy or disorganized space can activate their nervous systems and impact mental health wellness, claimed specialist Jesse Kahn, supervisor of The Gender & & Sexuality Therapy Center in NewYork If thats you, taking time to clean up your space can be an act of self-care and self-love, and may feel healing rather than like a chore you dont want to do.

Mindlessly scrolling with your social media sites feeds for hrs at a time is not just a time suck, however is additionally connected to reduced self-worth, rest problems as well as an enhanced fear of missing out, or FOMO.

Social media and the internet is a great resource to connect, cultivate support and community, but it can also be a place of overconsumption, distraction, and numbing out to what we truly may need in our lives, claimed McKel Hill Kooienga, a signed up dietitian in Nashville, Tennessee, as well as creator of the website Nutrition Stripped.

The apple iphones Screen Time function, Androids Digital Wellbeing devices or applications like Moment can check your social media sites use as well as assist you cut down. Other methods that might serve consist of disabling particular press alerts, switching over to grayscale setting or concealing your most attracting applications in a folder thats out your house display.

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All you require is a pen as well as some paper to get going. Journaling can be a healing practice that aids you recognize believed patterns, overcome tough feelings, assess particular occasions or grow even more appreciation in your day-to-day life.

Sometimes I find it just as helpful as therapy and Im very pro-therapy; Im studying to be a therapist, claimed Lauren Donelson, an author as well as yoga exercise educator based inSeattle Journaling helps us externalize whats going on inside our heads, and it helps us to look at our thoughts more objectively.

Making an initiative to obtain the advised 7 to 9 hrs of high quality slumber can make a massive distinction when it involves your total wellness. Getting an excellent evenings rest on a constant basis uses advantages such as far better immune feature, enhanced state of mind as well as far better efficiency at the workplace. (If you require some ideas on just how to make it take place, we have actually obtained you covered.)

Maybe the self-care practice here is getting a certain number of hours a night, not exceeding a certain number of hours, getting to sleep by a certain time so youre able to wake up by a certain time or creating a ritual to help you calm your body, relax and go to sleep, Kahn claimed.

Practicing reflection is just one of the very best ways to bring back as well as reconnect with our body and mind, claimed Tamara Levitt, a Toronto- based reflection teacher as well as h ead of mindfulness at Calm.

As (writer) Anne Lamott said: Almost everything will work again if you unplug it for a few minutes, Lamott claimed. There is immense value in giving ourselves time and space to shift from doing mode to being mode. Meditation allows us to reconnect with the needs of our mind and body.

If you favor led reflections, you can take a look at the totally free variation of applications like Headspace or Calm, or discover video clips on You Tube. And, obviously, practicing meditation in silence is one more fantastic choice that does not cost a dollar.

At the very least once daily, otherwise even more, take a while to sign in with on your own. Pause to examine just how starving or complete you are, any kind of feelings you might be really feeling or check your body for locations of rigidity.

Simply asking yourself the question, How am I doing right now? is a gentle reminder to take care of yourself, Hill Kooienga claimed.

Malte Mueller by means of Getty Images

It could be dancing in your room to a fire playlist, doing squats in your living-room or taking part in a neighborhood yoga exercise course (which is usually much less pricey than a store health and fitness course).

However, if that still doesnt fit in your budget, there are many free online yoga videos on YouTube, Kahn claimed. One of my faves is Yoga With Adriene.

Texting as well as e-mail are practical types of interaction, however they do not please our deep demand for link in the means a lot more individual communications do.

Call a friend, take a walk with a colleague or cook dinner with a family member, Dahlen deVos said. Connecting with others we care for helps to shift us out of our heads, regulates our nervous systems and elevates our moods.

The needs of job, household as well as various other responsibilities use up the majority of our energy and time, leaving hardly any kind of space in our timetables for tasks we absolutely delight in. But taking time for our leisure activities also when we have a whole lot on our plate issues.

Most of us are too busy to make time for activities that are joy-filled and feel nurturing, Levitt claimed. Find a time each week to shut off your electronics, and engage in a hobby that rejuvenates your spirit; play music, write in a journal, take a cooking class. While electronics deplete us, our favorite activities nourish us.

During high-stress durations, we might go hrs or perhaps an entire day without taking a complete, basing breath if were not willful regarding it.

I like to take a few deep breaths in the morning and also throughout the day because it helps me to recenter and connect more with the present moment, claimed Jessica Jones, a San Francisco- based signed up dietitian as well as founder of FoodHeaven One strategy that I use to remind myself to do this is to take three deep breaths every time I go to the bathroom and wash my hands. Its easy, free and makes a huge difference in my daily stress levels.

Choose your reason, whatever it might be, and after that find out a method you can join in.

Engaging in altruistic acts and seeing our actions make a direct and positive impact in the lives of others is a surefire way to shift your mood and feel part of something bigger than yourself, Dahlen deVos claimed. This can help put our problems in context, or at least give us a break from stressors without numbing out.

Malte Mueller by means of Getty Images

Aim to place even more of your grocery store spending plan towards veggies as well as much less in the direction of ultra-processed junk food. Then, to up your consumption, reduced up some veggies at the start of the week as well as shop them in your refrigerator this way you can quickly get them when you require a treat or include a handful or more to improve your dishes.

Most of us are not consuming near enough whole foods let alone vegetables, which keep us nice and full because of prolonged satiety from the fiber, Hill Kooienga claimed. Vegetables nourish our physical bodies on a cellular level with fiber, minerals, vitamins, and antioxidants, and they can taste really delicious too.

Snuggle up alongside your companion, your youngster or perhaps your BFF.

Cuddling releases oxytocin, a feel good hormone, that also helps with reducing stress, claimed Lynsie Seely, a marital relationship as well as household specialist in SanFrancisco

Pets make fantastic cuddle friends, also. Plus, spending quality time with our fuzzy pals has actually been revealed to reduce anxiousness, anxiety as well as sensations of isolation.

If you dont have access to a pet, go visit adoptable animals at the local shelter, sign up to walk dogs for a service such as WAG or sip tea at a cat cafe, Dahlen deVos claimed.

We typically consider self-care as doing something additional for ourselves in addition to our typical daily tasks. But self-care can additionally have to do with what you pick not to do, Seely claimed.

One means to offer a healthy and balanced no? Start setup limits with individuals in your life.

So many of us are people pleasers and spend a lot of time doing things out of feelings of guilt and obligation, causing us to feel energetically drained and lacking the ability to focus on ourselves and what we truly want, claimed Sara Groton, a nourishment as well as consuming psychology instructor in SanFrancisco Any time I discover myself assuming I ought to do that or I have to do that, I take a minute to concern as well as obstacle that believed.

All the face masks, manicures as well as massage therapies on the planet can not reverse the damages of that extreme inner guide slamming, evaluating as well as scolding on your own all day.

If you do not understand where to start with self-compassion, Allison Hart a psychological wellness expert in San Francisco advised placing your turn over your heart as well as stating to on your own: I am struggling right now. Im in pain, Im angry or feeling out of the flow. May I be gentle and flexible with myself. May I be kind to myself and may I take a break from problem-solving just for a moment.

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16 Ways To Practice Self-Care That Cost Next To Nothing - The Union Journal

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Catherine Burns Food4Thought

Published Feb 14, 2020 at 8:00 am(Updated Feb 14, 2020 at 12:30 am)

Bedroom benefits: adequate sleep improves everything and your diet can boost your sex drive

When I was 14 years old, I was given flowers on Valentines Day and they were beautiful.

For a few days, I existed in a bit of a spin wondering who this secret admirer could be. It turned out later that the flowers were from my stepdad, a clever move designed to improve my mums Valentines Day by not having a nobody-loves-me teenager in the house. (Genius, yes?)

By the time I found out, the world had moved on and I was too busy focusing on some other drama to be devastated. Decades later, theres no doubt that the day itself can be a little crazy. Hallmark have kinda run away with the concept, after all. But if you ask me, its always fun to focus on love!

Whether its cuddles on the sofa or bedroom Olympics, it helps to be in the mood. Thing is, many of us are so busy and stressed that our libidos tend to dive-bomb. Its hard to feel sexy when you still have to load the dishwasher and take out the trash. With work e-mails constantly pinging on our phones, toddlers reappearing long after bedtime and laundry coming out of our ears, we could be forgiven for choosing comfy pants and an early night.

So how, when we know in theory we would like to spice things up, do we actually make it happen?

Of course, clearing relationship grudges and confusion is always a good place to start. But chat to your friends or a therapist about that I wont pretend its my speciality area!

Where I can help you is with some nutrition tips that are surprisingly effective. What you eat and drink, likely has much more of an impact than you think. Read on!

Its not just what you do, its what you dont do

As promising as some supplements may be, you cant out-supplement a bad diet. If your libido is on the floor and you generally consume a processed diet full of junk, then no wonder. Theres no point taking zinc and essential fats with a burger and fries! Try and commit to a few weeks of clean eating and see how you feel on the other side. You might just feel so good that it motivates you to continue. And when you make dietary changes because you want to (not because you have to), then you have hit lifestyle gold. Its so easy!

Basic essentials

I mention zinc and essential fats for a reason. Both are important for healthy hormone balance, which is the foundation for a cracking sex drive. You can find zinc in pumpkin seeds, oats, oysters, turkey and shrimp. You can find essential fats in unroasted nuts and seeds, wild salmon, oily fish and avocados. If you dont like oily fish, then try a good quality fish oil supplement such as Nordic Naturals (Peoples), Life Extension (Rock On) or Innate Choice (Inside Out Wellness). If you are on medication for epilepsy, then fish oil supplements are usually contraindicated. This may be true for other meds (especially blood thinners), so always check in with your physician.

Balance your blood sugar

Key for hormone balance and mood. Also key for preventing the cravings that lead to sugar/junk food overload, which in turn leads to feelings of shame and regret, ie not feeling sexy! Try eating little and often through the day, only picking healthy carbs (eg wholegrains, root veg, fruit or beans/lentils) and always pairing those carbs with protein. A sample day: breakfast: vegetable omelette and a bowl of berries; snack: an apple and some almonds; lunch: salad with salmon or chicken, beans and olives; snack: carrots and hummus; dinner: quinoa with fish and green vegetables. If you need a little chocolate in your life, try some dark chocolate and a handful of nuts.

Stay hydrated

Its basic, but so important. The first sign of dehydration is fatigue. So if you dont have any energy, check you are drinking enough water. It also helps to prevent constipation, which can be the root cause of gas and bloating (a bit of a passion killer, lets face it).

Try collagen

Collagen is famous for improving skin health and elasticity including, (dont be shocked) the integrity of the vaginal wall! Pick a grass-fed collagen hydrolysate (eg the Great Lakes brand at Miles) and stir in 1 teaspoon at a time into tea or coffee. Goes fine in smoothies too. Its also good for phase 2 liver detox, gut health, hair growth and preventing wrinkles/cellulite. (Dont worry, the cashiers wont judge you although they might give you a wink!)

Sexy foods

Its not just oysters a good job, as I think theyre gross (each to their own!) but also bananas, watermelon, dark chocolate and high-quality protein can boost serotonin and your libido. Hooray!

Fenugreek

Often used to help improve breast milk production, fenugreek can also improve sex drive in men as it seems to regulate poor testosterone levels. Bumper breast milk supplies in women and an improved libido in men might lead to a somewhat bizarre or wet Valentines experience but . good luck! You can get fenugreek in supplement form or as a tea.

Sleep

Remember, adequate sleep improves everything. Specifically, though, men who only get four hours of sleep a night have the same testosterone level as men a decade older. Aside from that, we all need adequate rest for repair, memory, immunity and our sanity. So switch off Netflix and get into bed! In fact, try and ban all screens from the bedroom. If youre wondering how on earth you will wake up in the morning, then buy an actual alarm clock they do still exist!

I hope that helps! Looking forward to lots of November babies! Have a great weekend one and all.

Catherine Burns is a qualified nutritional therapist. For more details: natural.bm, 505-4725, Natural Nutrition Bermuda on Facebook and @naturalbda on Instagram

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Get in the mood for love! - Royal Gazette

Recommendation and review posted by Bethany Smith

- Salvador Rico, M.D., Ph.D., named Chief Medical Officer

- Martin Moorhead, Ph.D., promoted to Chief Technology Officer

SOUTH SAN FRANCISCO, Calif., Feb. 11, 2020 /PRNewswire/ --Encoded Therapeutics, Inc.(Encoded), a precision gene therapy company,today announced the appointment of Salvador Rico, M.D., Ph.D., as chief medical officer and the promotion of Martin Moorhead, Ph.D., to chief technology officer. Dr. Rico joins Encoded from Audentes Therapeutics, where he led clinical development of the company's pipeline of gene therapies for neuromuscular disorders. In his three years at Encoded, Dr. Moorhead has guided the development of the company's technology platform for creating innovative AAV-based gene therapies. He previously led the development of clonoSEQ, the FDA-approved next-generation sequencing assay for detecting minimal residual disease in lymphoid malignancies, at Adaptive Biotechnologies.

"Sal is an accomplished physician-scientist with deep experience advancing novel therapeutics through clinical development, and Martin is a strong leader who brings a genomics mindset to all aspects of gene therapy development," said Encoded co-founder and chief executive officer Kartik Ramamoorthi, Ph.D."With these appointments, we now have some of the most qualified gene therapy experts in the industry with a proven track record of delivering for patients in need. Their collective experience includes bringing multiple AAV-based gene therapies through clinical development, FDA filings, and approval. I am more confident than ever that our novel gene therapies can make a major impact on patients suffering from debilitating diseases, starting with Dravet Syndrome."

At Encoded, Dr. Rico will lead medical strategy and clinical development of ETX101, which is being developed for patients with SCN1A+ Dravet Syndrome. Dr. Moorhead will lead the technical team that enables Encoded's innovative research platform.

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"I am delighted to join an organization that is so committed to transforming patients' lives with the development of next-generation gene therapies," said Dr. Rico. "I look forward to working closely with both the team at Encoded, and with the Dravet Syndrome community, to advance ETX101 through clinical development and ultimately, deliver it to patients in need."

"In building a technology platform that combines the power of genomics and computation with AAV-based gene therapy, Encoded is forging the path for the next generation of precision genetic medicines," said Dr. Moorhead. "I am very proud of what we have accomplished to date and am thrilled at the opportunity to help advance multiple programs for diseases where no treatment options currently exist."

New Leadership Team Appointments

Salvador Rico, M.D. Ph.D.: Dr. Rico brings over 15 years of clinical research experience in the biopharmaceutical industry. Prior to joining Encoded, he served as senior vice president of clinical development at Audentes Therapeutics, an innovator in neuromuscular gene therapies that was recently acquired by Astellas, Inc. While there, Dr. Rico led the clinical development of AT132, an AAV-based gene therapy for X-Linked Myotubular Myopathy, participated in the design, conduct, and analysis of Phase I-IV clinical trials in multiple therapeutic areas, and successfully contributed to the FDA approval of multiple drugs, biological products, and medical devices including the INTERCEPT Blood System, Intermezzo, Hibor, Champix, Prolia, Kerydin, and others. Prior to joining Audentes, he led the clinical development teams at Cerus Corporation and Transcept Pharmaceuticals and was an investigator at the Centre for Drug Research, Hospital de la Santa Creu i Sant Pau in Barcelona, Spain. Dr. Rico earned his Doctor of Medicine and Surgery degree from the National Autonomous University of Mexico and holds an M.S. and a Ph.D. (Summa Cum Laude) in pharmacology from the Universitat Autonoma de Barcelona. He did his postdoctoral training in clinical pharmacology and transfusion medicine at the National Autonomous University of Mexico, and in pharmaceutical medicine at Universitat Autonoma de Barcelona.

Martin Moorhead, Ph.D.: Dr. Moorhead is a seasoned biotechnology executive with expertise that spans biology, assay development, machine learning, algorithm and software development. Prior to joining Encoded, he served as senior vice president of research and development at Adaptive Biotechnologies, and before that, as vice president of computational biology and informatics at Sequenta, Inc. Earlier in his career, he held positions at Affymetrix, ParAllele Bioscience, Viaken, and Synomics. He earned his Ph.D. in elementary particle physics from the University of Oxford, holds an M.Sc. in computing from the Imperial College of London, and earned his B.Sc. in physics from the University of Manchester. He also completed a postdoctoral research fellowship at the Lawrence Berkeley National Library, where he developed machine learning algorithms for data analysis.

About Encoded

Encoded Therapeutics, Inc., is a biotechnology company developing precision gene therapies for a broad range of severe genetic disorders. Our mission is to realize the potential of genomics-driven precision medicine by overcoming key limitations of viral gene therapy. We focus on delivering life-changing advances that move away from disease management and towards lasting disease modification. We are advancing our lead asset, ETX101, for the treatment of SCN1A-positiveDravet Syndrome. For more information, please visitwww.Encoded.com.

Media Contacts

Sarah SuttonGlover Park Groupssutton@gpg.com 202-337-0808

Danielle CanteyGlover Park Groupdcantey@gpg.com 202-337-0808

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SOURCE Encoded Therapeutics, Inc.

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Encoded Therapeutics Expands Gene Therapy Leadership with Key Appointment and Promotion - Yahoo Finance

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NEW HAVEN Death is not final in the Yale New Haven Hospital Pathology Department.

By going beyond the standard autopsy to examine a deceased patients DNA, the team led by Dr. Jon Morrow can find out the cause of an unexplained sudden death, including newborns, and inform families of inherited diseases that could help them make decisions about their health care.

As our knowledge of the genome has expanded there has increasingly been identified a number of genetic changes that have turned out to be causal in many of these cases of sudden death, said Morrow, chief of pathology for the hospital.

Yale New Haven is one of the few hospitals to do the intensive postmortem examination, and Morrow said they are interested in performing molecular autopsies on anyone whose family is willing to have one done or who has requested it before death, Thats because every exam adds to the body of knowledge about how genes contribute to disease.

It builds a database that helps us understand disorders at a deeper level than weve been able to do from normal autopsy, Morrow said.

Morrows team collaborates with the Yale School of Medicines Center for Genomic Health, which launched the Generations Project, which began in September with a goal of sequencing the DNA of 100,000 patients.

In a molecular autopsy, the pathology team looks at the exome, the 3 percent or less of the human genome that includes all of the genes involved in producing proteins. That 3 percent is responsible for everything you see in the body, Morrow said, because our organs are all made up of proteins. Its a very efficient way to look at almost everything thats important, he said.

The rest of the genome is involved in cellular structure, regulatory processes, how and when genes are switched on and off.

By examining the exome, pathologists can compare the individual to a reference database at the National Institutes of Health. The individual differences, called polymorphisms, are what can tell the doctors whether a patient who died from cancer did so simply because he smoked or because he had a genetic predisposition.

You always run what you find against this reference database, Morrow said. You can expect up to 70,000 variations in an individual. Most of those variations are innocuous. They dont have any real or deep pathologic significance.

By sequencing the genome, variations in alleles the pairs of genes that control characteristics will be identified and, if you find a variation in a patient that is extremely rare, lightbulbs go off, Morrow said. If that was a variation in a gene thats rare and a gene is known to control cardiac function, you would be very suspicious that that had to do with a cardiac malfunction.

In addition to finding the cause of death when a regular autopsy does not, Morrow said molecular autopsies have a larger goal of finding answers on the genetic level. He and his staff hope many people will consent to the procedure. Now, 12 to 14 percent of deceased patients undergo autopsies.

By looking at this exome, we get a pretty good view of what should be right. Theres wide variations in individuals, Morrow said. Some are pathologic, disease-causing. By moving beyond sudden death and extending these studies to all individuals now, we have a hope of explaining causes of all these conditions.

When word got out about Yale New Havens program, We had almost universal enthusiasm from a number of major medical centers [asking], Can we partner with you? Morrow said. Im hopeful wed be able to expand this to multi-institutional practices.

Some diseases, such as many cancers, start with genetic mutations that are caused by external factors, such as smoking. Those are called somatic mutations. What the molecular autopsy finds are inherited mutations that may give a predisposition to the cancer.

Were not sequencing tumors, were sequencing normal tissue, Morrow said. When we find a damaging lesion, that gene was present from birth and that gene was inherited.

Another example is alpha-1 antitrypsin deficiency, an inherited disorder that is responsible for both lung and liver disease. Its not that uncommon in the population and people get emphysema and liver disease later in life because of it, Morrow said. Finding out that a deceased relative had the disorder can help family members lessen their risk, by not smoking, for example.

Finding a genetic cause of death is especially valuable in fetal deaths that have no anatomic cause of death, he said. For a lot of these mothers, just reading their notes, they have a lot of guilt. So if we can identify through whole-exome sequencing, it would at least give them something. As many as 30 percent of stillbirths and infant deaths may be genetically caused, Morrow said.

They want to know sometimes, am I at risk? said Amanda Masters, a pathologists assistant on Morrows team. If we do find a genetic link, if it was inherited, it could help them and future generations.

Some families do not want the autopsy done, and a few consent to it but dont want to know the results, Morrow said. But almost all do want to understand how their relatives DNA may affect their health.

The hospital has agreed to provide one genetic counseling session free of charge to those families, Morrow said, adding that the results of the autopsy are sent to the genomics lab. Decisions are discussed. Do you need to be tested? You have to be very careful because you never want to send up false flags. Were very conservative in the way we approach this.

That is important because even genetic testing isnt infallible. When the genomic revolution started there was overpromising of what it could tell you, Morrow said. Besides wasting money on genetic testing, it also caused angst.

Often, even after a standard autopsy, we still dont know the reason why they died, even with slides and looking at tissue at a very, very close level, said Dr. Keith Churchwell, executive vice president and chief operating officer of the Yale New Haven Health System. The lack of information leaves a real void, he said.

Conducting a genetic examination of a deceased patient will reveal information for the next generations, for that family and for their children, Churchwell said.

Theres three aspects of doing this that I think are good, Morrow said. One is it helps us move the autopsy from what weve been doing into a new realm that helps get deeper answers into disease causation and relevance into that diseased individual. It offers a pathway for more rational decision-making for individuals related to the [deceased patient].

The third thing, which goes back to the Generations Project, [is] whether we find anything relevant or not to the disease. It provides a growing database that we can correlate the variations into, Morrow said. For example, doctors might discover a liver disease of a certain type that we might not know before.

It is a wonderful platform for future discovery on genetic changes, he said. Its a huge task to figure out which variations are meaningful.

Morrow said people who are concerned about privacy, worried that their life insurance rates will rise or that theyll lose their job, shouldnt be. Since the autopsy is done on a relative, and the medical record is protected by federal privacy laws known as HIPAA, the information wouldnt appear on a relatives record anyway. In a way its the perfect vehicle for getting some information at almost no risk, he said.

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Autopsies at Yale New Haven Hospital find cause of death in genes - West Hartford News

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