An increasingly common question I get asked is, Will gene testing help my doctor know which antidepressant to prescribe? Popular tests such as GeneSight suggests that they can shorten your road to recovery and how you, as an individual, will respond to specific antidepressant medications.

Does drug-gene testing, also referred to as pharmacogenomics or pharmacogenetics, work? And if so, does it only work for certain types of medications? Lets find out.

The idea of gene-drug testing is pretty simple. By testing your DNA, companies hope to be able to predict your response (or likely non-response) to specific types of antidepressants. Its also being marketed for a number of other diseases and medications.

Just a year ago, GeneSight had some pretty strong marketing language on its site. The company was strongly suggesting its test could help your doctor choose the best antidepressant for you:

Fortunately, the GeneSight genetic test can provide doctors answers that quickly lead to relief. Pharmacogenomic testing helps empower your doctor with the exact information needed to prescribe you the best medication for you. By examining how your DNA responds to specific medications such as antidepressants, this simple, painless test lets doctors know which medications may not work for you, so you can get back to feeling like yourself again. [] Through pharmacogenomic testing, your doctor can identify the correct medication and create a personalized treatment for you.

In the 2018 announcement of its own antidepressant test, another gene-drug testing company called Color says it now analyzes a number of these genes, starting with two that can impact your response to certain mental health medications like Zoloft, Paxil, and Lexapro. The blog entry cites seven research studies, but none of them have anything to do with antidepressants.

Few genetic researchers feel as positive about the current usefulness of gene-drug testing than companies marketing these tests. The American Psychiatric Associations research council reviewed the evidence last year and found that such genetic testing is not really ready for mass consumption.

Greden et al. (2019) looked at using pharmacogenomics directly to help in depression treatment. Because the researchers didnt find a significant difference (either statistically or clinically) in their primary outcome measure, they instead emphasized the statistical significance they found in two of the 25 secondary outcome measures they examined.

In treatment research, scientists increasingly use a statistic called Number Needed to Treat (NNT) that allows for cross-comparisons of the real-world efficacy of different kinds of treatment. The National Institute for Clinical Excellence (NICE) in the UK recommends that for a treatment to be clinically significant, the NNT should be in the single digits.

According to a critique of the researchers (Goldberg et al., 2019), the Greden study had an NNT of 17 for a response to an antidepressant and an NNT of 19 for remission of a depressive episode. Not exactly powerful numbers. In fact, combined with the non-significance of the primary outcome studied, Greden ironically demonstrated that pharmacogenomics doesnt appear to very good at its primary goal of helping to guide antidepressant treatment.

In short, the science today doesnt support the mainstream use of these tests for antidepressants.

Personalized medicine is the new New Thing marketed by anyone who has access to a DNA lab. The problem is that the marketing of gene-drug testing far overshadows the science. In early 2019, the U.S. Food and Drug Administration updated its guidance on gene-drug testing:

[The] FDA is aware of genetic tests that claim results can be used to help physicians identify which antidepressant medication would have increased effectiveness or side effects compared to other antidepressant medications. However, the relationship between DNA variations and the effectiveness of antidepressant medication has never been established. []

Do not change or stop taking any medicine based on a report from a genetic test you took on your own. []

[And to doctors:] If you are using, or considering using, a genetic test to predict a patients response to specific medications, be aware that for most medications, the relationship between DNA variations and the medications effects has not been established.

Goldberg et al. (2019) said it best:

[Researchers] have noted that commercial [] test manufacturers promote their products with a zeal that is disproportionate to the existing evidence base particularly when marketing to the lay public and clinicians who are likely unfamiliar with the limited statistical power of candidate gene association studies.

Youd be wasting your money by purchasing one of these tests in hopes of getting better results from your antidepressant treatment. The science simply doesnt support use of these tests at this time.

Online health information isnt always accurate on this issue even from trusted sources. For instance, the Mayo Clinic suggest these tests can help, but its unclear whether the anonymous, unlisted author of that article has examined the primary research (as there are no research references listed in the article). Harvard Health Publishing, on the other hand, got it right by noting that the research of gene-drug testing showed no evidence of effectiveness.

Someday, the hope is that pharmacogenetics may meaningfully inform treatment decisions, as it does in oncology. But were not yet there.

References

Goldberg, J.F., Rosenblat, J.D., McIntyre, R.S., Preskorn, S.H., de Leon, J. (2019). Letter to the Editor: Clinical versus statistical significance of pharmacogenomic-guided antidepressant therapy: Whats really being measured and marketed? Journal of Psychiatric Research, 114, 208-209.

Greden, J.F., Parikh, S.V., Rothschild, A.J., et al. (2019). Impact of pharmacogenomics on clinical outcomes in major depressive disorder in the GUIDED trial: a large, patient-and rater-blinded, randomized, controlled study. J. Psychiatr. Res. 111, 5967. https://doi.org/10.1016/j.jpsychires.2019.01.003

Related Articles

Read more:
Gene Testing for Antidepressants & Psychotropics: Not There Yet - PsychCentral.com

Recommendation and review posted by Bethany Smith

The public called it bubble boy disease. Until recently, any infant born with this rare genetic disordera mutation called severe combined immunodeficiency (SCID-X1), linked to the X chromosomehad little chance of surviving outside a small, sterile environment. Brian Sorrentino, a hematologist and gene therapy researcher at St. Jude Childrens Research Hospital in Memphis, Tennessee, made it his lifes mission to cure this disease.

Sorrentino died at 60 in November 2018, before the groundbreaking results of the first trial were published in The New England Journal of Medicine. As a teenager, Sorrentino had been treated for Hodgkins lymphoma, with heavy doses of radiation. This led to various other ailments later in life, including heart disease and then terminal lung cancer.

He felt like there was a reason that he was saved at 17, says his widow, Suzanne Sorrentino, who also lost her first husband to lung cancer. The work he did to save patients with bubble boy disease was it.

The team thats now carrying on Sorrentinos work at St. Jude won the 2019 Smithsonian magazine American Ingenuity Award in the life sciences category. We spoke with his widow to learn more about the man who started it all. A condensed interview is below.

Can you tell us a bit about your husbands background and what brought him to St. Jude?

Brian was from New York. His dad was a radiologist, and he thought being a doctor would be the greatest profession in the world. After medical school, he worked at the National Institutes of Health with Dr. Arthur Nienhuis. When Dr. Nienhuis came to Memphis to be the head of St. Jude, Brian came with him. That was in 1993. It took Brian some time to adjust to the South. During the years I knew him, we were still working on getting him to say yall instead of you guys.

He was divorced, and the father of two grown children, when I met him just over five years ago. Id never met a scientist before. I told him, I just picture you in a lab coat looking at a microscope all day. He told me that was part of it, but it was a lot more than that.

Was he able to experience the joy of seeing his work on SCID-X1 come to fruition?

Oh, yes. When they got started with the trial, he was so excited. He and [fellow St. Jude researcher Dr.] Ewelina [Mamcarz] were just giddy when they had some children enrolled.

Its one thing to be in your lab and think youve got it. But to go over and see the child and see the parents who are just desperate, it made it real. He said some of the greatest days to him were when he got to leave his lab, his part of St. Jude, and go over where the patients are.

When Brian died, they had treated 10 patients, and theyd come from all over the world. In one of my favorite pictures of him, hes holding one of the children and hes got the biggest smile on his face.

Whats it like to watch the legacy of his work unfold and get celebrated?

Its bittersweet. Brian would say, Recognition is really nice, but its not important. Whats important is the science and saving these children. He didnt live to see the paper about the trial published in the New England Journal of Medicine, but he did know that it had been accepted.

When the paper came out in April, there was so much hubbub about it. The St. Jude PR department was just overwhelmed. They thought it would be big, but not as big as it turned out to be. I think Brian would have been a little embarrassed. St. Jude had a symposium to honor Brian and one of his colleagues in June. It was really lovely, but I just think, Damn it, he should be here!

Beyond scientific research, what were some of Brians other passions?

He played guitar. At a big, nice, fancy St. Jude dinner, with everybody all dressed up, he got up onstage and played Mustang Sally with the band that was performing. He was so nervous. I got so sick of that song. Hed played it a thousand times before the dinner because he didnt want to mess up in front of his colleagues.

He loved the Grateful Dead, which I never understood. I went to four Grateful Dead concerts with him, and told him, You owe me. This music is awful. I like music that has a beginning and an end. He would try to explain how wonderful it was. We buried him in a Jerry Garcia T-shirt.

He also loved his Corvette. He drove it on weekends, and that was his fun car. He had a Volkswagen that he would drive to work that he called his beater. He would get in that beater and drive like he was 85 years old. Hed get in his Corvette and go 100 miles an hour. At the symposium St. Jude had to honor him, everybody who spoke, including the head of the hospital, got up and had some horror story of riding with Brian in his Corvette.

He was just witty and wonderful. And there was a whole new world for me when I met him.

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Honoring the Legacy of Brian Sorrentino | Innovation - Smithsonian.com

Recommendation and review posted by Bethany Smith

Twelve-year-old Ash Lammin, who was born a boy, is one of Britains youngest transgender children (Picture: KMG /SWNS.COM)

Ash Lammin says she felt that she was a girl as soon as she could speak.

The 12-year-old, from Kent, says that it has been difficult growing up as a transgender girl but feels that she is firmly on the right path.

Now Ash is beginning to transition her gender from male to female at an NHS-run clinic and is one of the youngest children in the country to do so.

Ash who changed her name by deed poll to Ashley when she was eight will start by taking hormone blockers to halt the onset of puberty.

She eventually wants a womb transplant so she can be a mother when she is older.

She said: The journey is long and its still going, but I feel like the sense of victory is there through it all. I do feel accepted sometimes, but other times not.

Not everyone is going to understand and people have to have their own opinions and I understand that. Some people might not like the idea of trans.

I hope I inspire others but I just hope that love and acceptance comes through everything.

Mum Terri has said that Ash will take the blockers until she is 18. At that point, she herself will decide whether to go ahead with gender reassignment surgery.

If she decides not to go ahead with it, Ash will come off the blocker, and her puberty will kick in just a few years later than her peers.

She said: I never thought it was a phase, Ash was just Ash. When she was three she said to me, Im a boy because you gave me a boys name its your fault.

I remember feeling horrible, because she blamed me.

Id never come across it before and I just went along with it. I just thought at the time if hes happy, well thats the main thing.

When Ash turned 11 and went to secondary school, she became a target for bullies who would throw things at her on the bus and shout abuse at her, forcing Terri to take her out of the school after just one term.

Ash is now being home-schooled and Terri is calling for better education within schools to teach children about transgender people.

She said: Id like to see the subject of transgender people included in some lessons, like there are about same-sex families.

There needs to be more about liking people for who they are, not what they are.

MORE: Transgender teen has penis farewell party before gender reassignment surgery

MORE: Transgender man is asked to go as a bridesmaid to mothers wedding despite identifying as a guy

MORE: Transgender teen has penis farewell party before gender reassignment surgery

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12-year-old transgender preteen has started transitioning - Metro.co.uk

Recommendation and review posted by Bethany Smith

December 10, 2019 Cover

Off-road racing has always been a big part of the Laughlin event scene. Drivers of heavy duty racing trucks and buggies like the challenge of negotiating hairpin turns and switch-backs over a rugged desert course, daring them to take their skills to the next level which is why the 2019 McKenzies Rage at the Rivers season finale is a popular event.

When it comes to portraying Johnny Cash, tribute artists dont get much closer than Shawn Barker.Cash commanded and earned the kind of serious respect that continues long after his passing, and Barker has kept that respect at the very core of everything he does from the very beginning. Sure, an artist might include the kind of fun Cash used to have, because they know full-well his fans expect nothing less.

If anyone knows about the healing power of laughter, its Will C, a.k.a. William Clifton, because hes seen it happen.The veteran of both comedy and American armed forces, Will C lives by his message that laughter is truly the best medicine.On a dare he took the stage at the World Famous Comedy Store in La Jolla, California, in the spring of 1995. He caught the comedy bug and never looked back.

A sip of Cella wine is all it takes to taste the heart and soul that goes into crafting each blend at the Kingman winery and vineyard.For Carlos Cella, owner of Cella Wines, the craft is his passion, and it is evident in the high-quality flavor and aroma that bursts from every bottle.Cella has been making wines since childhood with his Italian upbringing.

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Reviews of female viagra - Fox news female viagra - Is there a female viagra yet - Laughlin Entertainer

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Many of us biologists conduct fieldwork in diverse places, from Alaska to the tropics, from aiming to understand how microbes are responding to climate change in the boreal soils to learning about life history strategies and co-evolutionary arms races of bats, their ectoparasitic flies, and the ectoparasitic fungi living on those flies.

The days before fieldwork tend to be hectic: make a checklist to make sure you have everything you need, think about a plan B (and a plan C, just in case), anticipate drawbacks and plan on how to address them, and the list goes on and on. The day comes. You make it to your field site, you collect the samples you want, obtain the data you need, everything works out just like planned, and you make it back to the lab safe, on time, and without going over your planned budget. This is how it should be, but it never really goes like that.

Fieldwork is one of the most exciting experiences about doing research. It is also, in many cases, high-risk. During fieldwork, many things can go wrong, and most of those things cannot be helped. We cannot control the appearances of massive puddles in the middle of the road, critically damaging our transportation vehicles. We cannot control the thunderstorm that makes our study organisms disappear when we finally arrive at a remote field site after hours of climbing a mud-covered mountain.

Sadly, this is not always the case for threats to our integrity as human beings, and we, as a scientific community, have done far too little to address this problem. People from underrepresented groups in the sciences such as people of color, women, and those who identify as LGBTQIA+ or gender nonconforming often are at higher risk of suffering abuse during fieldwork. This comes in the form of sexual harassment, sexual abuse, discrimination, and intimidation. Scientists who have experienced abuse often fear talking about it because they are traumatized and because they fear retaliation and backlash, especially if the perpetrators of abuse are colleagues or superiors advisers and people at higher career stage.

In Spring 2018, we carried out an anonymous survey to collect testimonies of what scientists, specifically from the LGBTQIA+ community, experience during fieldwork. The idea for such a survey sprouted from concerns that sexual orientation or gender identity may play an unwanted or unwarranted role in peoples professional career. Especially during fieldwork, when Diversity and Inclusion Offices from our university campuses are far away, LGBTQIA+ researchers are exposed to people who may not agree with their sexual orientation or who do not understand why he may want to be addressed as they.

Responses revealed experiences ranging from discrimination to situations that made researchers decide to no longer perform fieldwork outside of safe places. This adds a whole new level to fieldwork stress, namely having to evaluate sites for their tolerance towards LGBTQIA+. In one story from fieldwork, men voiced discomfort because an openly gay man would share a room with them while, simultaneously, women felt uncomfortable due to the possibility of having to share a room with someone from the opposite sex. Another survey respondent described that they were fearful to carry out fieldwork in places that are recognized for their homophobic culture. These experiences leave people feeling isolated and rejected.

We present a few strategies that we can instill in STEM fields to avoid cases like these:

1) INFORM PEOPLE ABOUT LGBTQIA+. Erase any misinformation that may exist. For example, a gay man is not a threat to the sexuality of cisgender males. Institutions can facilitate trainings on diversity and inclusiveness and provide information on the LGBTQIA+ community to eliminate negative stereotypes.

2) HAVE SUFFICIENT FUNDING AVAILABLE FOR FIELDWORK. Although sometimes it's unavoidable to share rooms due to limited budget or space, if there is the possibility to do so, provide individual lodging for people traveling to fieldwork or conferences. Especially for those who ask for it.

3) DEVELOP AN EMERGENCY PROTOCOL. As a lab, department, or institution, develop a protocol that scientists can follow as a response to experiencing a threat to their integrity. Protocols like this should be part of a broader departmental or university-wide mission statement about equity in field work. The bar has been set high by this example of a mission statement written by University of California Irvine professor Kathleen Treseder.

4) AVOID INTOLERANT AREAS. It is important to note that this does not only apply to countries like Niger and Tunisia where discriminatory laws expose LGBTQIA+ individuals to the risk of death penalty. It also applies close to home, in the USA, where there is an ongoing debate about public restrooms and which one transgender people and people who identify as gender-nonconforming should use.

5) IMPLEMENT A ZERO-TOLERANCE POLICY. Inform everyone in your lab, department and institution that there is a zero-tolerance policy regarding abuse. A code of conduct with expected versus unaccepted behavior and practices should always be made available through trainings and in field stations.

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Your Apple smartwatch may be the key to detecting heart issues before they happen - Massive Science

Recommendation and review posted by Bethany Smith

GLEN MILLS, Pa., Dec. 10, 2019 /PRNewswire/ --SmartSolutions RX, Inc. announces the launch of a new scientifically based, drug-free hair support system LOCKrx, which includes both ingestible and topical Healthy Hair Programs that create the ideal environment to maintain hair follicle cycle and growth.

Hair thinning and hair loss are a pervasive problem, affecting an estimated 80 million men and women in the U.S. Smoking, diet, stress, environment and genetics all contribute to hair loss, as well as the hormone DHT which shrinks the hair follicle and is the primary cause of loss in male and female pattern baldness. The current treatments often come with unwanted side effects and take months to generate results.

LOCKrx is a drug-free hair treatment system that addresses scalp health, both internally and externally, that directly impacts hair growth and quality.

"When formulating LOCKrx, we meticulously designed and tested both ingestible and topical ingredients that are scientifically proven to reduce the inflammation associated with damaged hair follicles and thereby improve quality of the hair," said Cynthia Rager, President and COO of Vision Medical, Inc. "Our topical LOCKrx solutions include specific growth factors clinically shown to play a key role in the hair follicle growth pathway as well as to enhance wound repair and skin regeneration, all of which improve scalp skin health, while also playing a vital role in the proliferation of skin and hair cells."

LOCKrx is available as both internal and external treatment plans, designed to work synergistically to promote healthy scalp skin and hair growth.

"Growthfactors possess the ability to stimulate hair growth through variousmechanistic pathways," said Richard Jin, M.D., Ph.D., Hair Regeneration Specialist, RJClinical Institute. "We have experienced very positive results when combiningthese with platelet rich plasma therapy to promote new and existing hairgrowthas well as using it as an alternative to PRP. This has helped us treat the mostcommon form of hair loss known as androgenetic alopecia, as well as increasethickness and density of hair in post-transplant patients."

LOCKrx Inside Healthy Hair Programis a 3-step, 6-week ingestible plan that uses unique marine and botanical ingredients, amino acids, and vitamins to address total body inflammation.

1. DEFENSE Gut Health - Prebiotic supplement that includes mineral-rich blue green algae and proven anti-inflammatory botanicals curcumin, aloe, licorice and beta-glucan to address gut health in powdered form.

2. BLOCK Hair Loss- Follicle-enrichment supplement formulated with the LOCKrx proprietary blend of botanicals, adaptogens, marine collagen, and saw palmetto to help support hair growth and block conversion of testosterone to DHT, one of the major causes of hair loss in male and female pattern baldness.

3. GUARD Healthy Hair Tabs- Premier blend of complexed Vitamin B plus biotin in the most bio-available form.

LOCKrx Outside Healthy Hair Programis a combination of clinical and at-home applications of growth factor solutions that support and balance the scalp microbiome, while enhancing the environment for healthy hair growth:

VisionMedical, Inc. has exclusive physician distribution rights for LOCKrx.

Smart Solutions RX, Inc.

Smart Solutions RX, Inc. formulates, develops, manufactures and distributes products for medical aesthetic applications to hair and skin. A blend of scientific research and innovative formulation and delivery systems are the hallmark, as evidenced in the LOCKrx brand for healthy hair support. Medical aesthetic protocols and workshops are integrated into the superior customer support program. http://www.smartsolutionsrx.com

Vision Medical, Inc.

Founded in 2013, VisionMedical, Inc. develops, manufactures, and marketsmedical and aesthetic technology for the medical and aesthetic marketsfor worldwide distribution.Vision Medical's first commercial product, theSmartGraft Hair Restoration System, incorporates an award-winning Automated Follicular Unit Extraction (FUE) system for men and women.Featuring theindustry's first closed harvesting system, SmartGraft allows physicians to harvestgrafts more efficiently while keeping grafts moist prior to implantation. http://www.SmartGraft.com.

CONNECT WITH US

Instagram: @SmartSolutionsRXTwitter: @SSolutionsXFacebook: @SmartSolutionsRXinc

Original post:
SmartSolutions RX Launches LOCKrx, A Drug-Free Support System Fighting To Keep Hair Thick and Healthy - Daily American Online

Recommendation and review posted by Bethany Smith

DUBLIN--(BUSINESS WIRE)--The "Global Cancer Diagnostics Market Analysis: Emerging Opportunities in the USA, Europe, Japan-Supplier Shares and Strategies, Latest Tumor Markers, Volume and Sales Segment Forecasts, Technology and Instrumentation" report has been added to ResearchAndMarkets.com's offering.

The new report is a study of the major business opportunities emerging in the global cancer diagnostics market during the next five years. The report examines trends in the U.S., Europe and Japan; reviews current and emerging assays; analyzes potential applications of new diagnostic technologies; forecasts sales of major tumor markers by country and market segment; profiles leading players and potential market entrants; and identifies specific business opportunities for suppliers.

Rationale

The cancer diagnostics market is on the verge of explosion, as the researchers approach major technological breakthroughs in tumor diagnosis and therapy, discover new specific antigens, and unlock the mystery of the genetic basis of the disease. During the next five years, the worldwide cancer diagnostics market is promising to be an exciting, dynamic and rapidly expanding field.

Anticipated technological breakthroughs will create numerous opportunities for determining genetic predisposition, detecting specific tumors, and monitoring biological response to cancer therapy. The rise in geriatric population will further compound the growing demand for malignancy assays and the rapid market expansion worldwide.

Worldwide Market Overview

Business Opportunities and Strategic Recommendations

Over 200 Current and Emerging Cancer Diagnostic Test

Supplier Shares, Sales and Volume Forecasts

Five-year test volume and sales forecasts for major tumor markers by country and market segment, including:

Instrumentation Review

Technology Assessment

Competitive Strategies

The companies analyzed in the report include:

For more information about this report visit https://www.researchandmarkets.com/r/vxuvz9

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Global Cancer Diagnostics Market Report 2019: Emerging Opportunities, Supplier Shares, Strategies, Latest Tumor Markers, Volume and Sales Segment...

Recommendation and review posted by Bethany Smith

The human immune system is highly complex and multi-faceted. Seemingly an infinite number of things can affect our internal landscape and alter how our immune system defenses respond to and fight foreign invaders to keep us healthy. The misconception a lot of people have is thinking that we want a super active immune system that is like Rambo, an ultimate killing machine! Really what we want is a balanced immune system, that neither swings into hyperactivity nor falls into decreased function. If our immune system functions too high, we end up with autoimmune disorders this is when the immune system attacks our bodys cells, not just foreign invaders, if it is not functioning as it should, when exposed to germs we get sick and have to fight off an illness.

Did you know that just the time of year can affect our immune system function? Just the fact that the daylight does not last as long can cause our immune system to be less responsive. Shorter days and colder weather cause people to stay inside more, reducing the amount of natural vitamin D they make, which is a key immune system booster. An alternative for the individual not wanting to go outside is supplementing Vitamin D into their diet.

See more here:
Seasonally Decreased Immune Function Around the Holidays - Matthews Beacon

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Disrupted sleep, irritability, sweats, rapid heart rate 67-year-old Nora Barler has battled extreme hot flashes for three decades, ever since having a total hysterectomy in her late 30s to remove her uterus and ovaries.

When her hot flashes began, she was working in human resources as a labor and employee relations manager and felt self-conscious when one would strike during meetings. I was in meetings with high-level executives, at times the only woman, and I would start sweating up a storm. Beads of sweat would be dripping into my contact lenses, burning my eyes and [running] down my face. My makeup would be smearing. I could feel my heart going boom, boom, boom, she recalls.

The hot flashes also regularly struck at night, despite Barler enlisting central air conditioning, a window unit in the bedroom and two fans. When they were really bad at night, I used cold packs I kept in the freezer. It sounds over the top, but thats how it often was. My husband was freezing, and I kept him awake. And because I didnt sleep, I was exhausted when I went to work. I was irritable and antsy.

Hot flashes during menopause are a fact of life for most women. They come on rapidly and then spontaneously resolve after a few minutes, explains Barbara Soltes, MD, a gynecologic endocrinologist at Rush University Medical Center.

[Women] feel a sensation of heat or a flush over the upper part of their bodies, which subsides in minutes and is followed by perspiration down their face, she says. It is associated with an increase in heart rate and skin temperature, which also subsides in minutes.

Hot flashes during the day can interrupt work or other activities. At night, they can disrupt sleep, which can kick off a cascade of complications: lower energy levels, lower cognition levels, fatigue and irritability. Hot flash symptoms can persist for seven to 11 years or more, according to the long-term Study of Womens Health Across the Nation (SWAN).

Treatment options include:

Estrogen replacement is very effective at easing hot flashes, but women should discuss hormone replacement therapy with their physician because of the risks. Research has found that women who used hormone treatments had a higher risk of cancer, heart disease, stroke and blood clots.

Estrogen can be valuable as a short-term treatment for women experiencing hot flashes and night sweats, according to the National Institute on Aging. Estrogen replacement therapy, which is approved by the Food and Drug Administration (FDA), comes as a pill, patch, gel, topical spray or vaginal ring. The pellet form is not FDA-approved.

Provided there are no significant risk factors, such as active heart disease, estrogen-dependent cancer or a history of blood clots, we will start at a low dose of bio-identical hormone replacement, Soltes says. After six to eight weeks, the dose may be adjusted.

Certain antidepressants in particular, selective serotonin reuptake inhibitors (SSRIs) prescribed in a low dose can help reduce hot flashes. They act by altering the brain neurotransmitters involved in temperature regulation. Low-dose paroxetine (Brisdelle) is the only FDA-approved SSRI for treatment of hot flashes.

Clonidine, a blood pressure medication, and gabapentin, prescribed for seizures and pain, are also used to relieve hot flashes, although they are used off-label.

If hot flashes are not severe, some women take the herb black cohosh or bee pollen, though research studies have been small and have had mixed results. There has been quite a bit of research on herbal therapies, most of which do not provide more symptomatic relief than a placebo, Soltes says. Black cohosh has been the only herb that seems to have estrogen-like qualities, which may be effective in providing relief for at least six months.

Weekly acupuncture treatments may also be effective, she says. Acupuncture has been shown to reduce the frequency of hot flashes, although it does not work for all women, according to a 2016 study from Wake Forest Baptist Medical Center published in the journal Menopause.

Lifestyle measures include avoiding caffeine, alcohol and spicy foods, adopting stress-reduction techniques and dressing in layers of cotton clothing, Soltes says.

David Walega, MD, associate professor of anesthesiology and pain management at Northwestern Universitys Feinberg School of Medicine, has been researching new treatment options.

His 2013 study of a numbing medicine, injected in the neck, showed promising results on a small sample of women. Half of the women received an injection of bupivacaine hydrochloride a local anesthetic that blocks nerves and can alleviate pain into the stellate ganglion, a nerve bundle in the neck. The other half received a placebo injection.

Women who received the real injection had a 52% decrease in their moderate to severe hot flashes at the six-month follow-up a statistically strong effect, Walega says. Secondarily, we saw trends of improved depression and anxiety scores and also observed improved verbal learning on cognition testing in the treatment group but no improvement in the placebo group, he says. The study showed that benefits of the injection lasted at least six months. In some cases, Walega has seen benefits last as long as 18 months.

Walega and his team are working on a similar study of the numbing injection with a larger group of women. He hopes that the results, expected in about three years, will give women a safe option to effectively treat their debilitating hot flashes.

Barler participated in Walegas study. She had tried hormone pills and hormone patches. The pills didnt work for her, and the patches were expensive, not covered by her insurance and had some cancer risks.

When nothing seemed to help, Walegas numbing injection did. It was a life-changer, Barler says. Although the pilot study has ended, she continues to get the injections and has gone as long as two years between injections.

Barler has changed from constantly being hot to being comfortable. I used to wear shorts and tank tops at home, even in winter, she says. Now I wear long-sleeve sweatshirts, yoga pants and warm socks around the house.

While hot flashes can still be debilitating to many women, the hope is that new research will take the heat off of menopause.

Nancy Maes, who studied and worked in France for 10 years, writes about health, cultural events, food and the healing power of the arts.

Excerpt from:
Hot Flashes Can Be Fast and Furious - Chicago Health

Recommendation and review posted by Bethany Smith

Sunday evening, Ryan T. Anderson and Robert P. George authored: Physical Interventions on the Bodies of Children to Affirm their Gender Identity Violate Sound Medical Ethics and Should be Prohibited. The outlet for this nonsense is Witherspoon Institute's pretentious blog.

I say nonsense because neither author is concerned for the welfare of gender incongruent children. Nor do they voice legitimate concern for medical ethics because ethics are predicated on medical science. Treatment of children should be in accordance with the best available evidence.

Doing so is ethical per se. Neither Anderson nor George care about the medical science. As you will see, it is what they propose that is unethical because it deviates from accepted medical practice.

This entire exercise is a pseudo-scientific effort to support the teachings of the Catholic Church. The term gender identity is within defense quotes because the Church teaches that gender identity doesn't exist. The Vatican has explicitly stated that Church teachings about gender are based on conforming with Genesis 1:27.

The next time that Anderson or George are ill I doubt that they will ask a physician to substitute information from ancient texts of dubious provenance for evidence-based science.

We begin with the verbose subtitle:

Gender identity is formed by the age of two or three. Parents and others treat toddlers with the assumption that their gender and natal sex are consistent. If children are subject to any influence it is cisgender affirmation. We teach children that they are what their genitalia depicts. Gender identity is independent of parental influence. They continue:

In other words, daddy turned his kid into an ATM.

The greater dishonesty is that this case has little to do with the child's medical care. At seven years of age, she will not be a possible candidate for medical interventions for several years. Puberty blockers might be appropriate at age 12 to 13. Whether or not she receives medications will be based upon the persistence of the condition. Persistence is a function of severity.

By the way, the judge who upended a jury verdict in favor of the kid's mother has been recused. Throughout this saga, mom was indifferent to the media knowing that less attention to this case favored the child. Dad, on the other hand, turned this case into a means of support. He was in continuous contact with conservative Christian media. Dad's behavior has been consistent with his history of dishonesty. But I digress.

Getting back to Andergeorge:

I agree with Andergeorge that ethics are an important consideration. Medical ethics are generally defined by the best available information derived from peer-reviewed research published to respected academic journals. That research is based on evidence.

Based upon the evidence-based science, the ethics of treating gender dysphoria in children are expressed by the American Academy of Pediatrics. The AAP's policy statement defines best practices as the gender-affirmative care model.

Deviating from that policy constitutes a breach of ethics. That is particularly true when the deviation is based upon faith over science. The only way that Anderson and George can support Church teachings is to turn a medical condition into an ideology. When either of them can cite peer-reviewed research to support their contention they might have a legitimate argument.

In other words, the following paragraph is, well baloney:

In children, gender dysphoria diagnosis involves at least six of the following and an associated significant distress or impairment in function, lasting at least six months.

Neither Ryan T. Anderson nor Robert P. George have the erudition to assess the science. They do cite some research dishonestly in order to quote:

With approximately one-third of TGD [transgender and gender diverse] adults and 40 percent of TGD youth identifying as nonbinary, care guidelines that reinforce binary systems of gender identity may limit access to clinical services and restrict the ability of nonbinary people to navigate medical systems. Framing gender as solely binary defines therapeutic options and outcomes only in reference to two gender experiences, which impacts access.

They mention the informed consent model. This was developed at the Fenway Institute in Boston, at least in part with the guidance of Dr. Sari Reisner at Harvard Medical School. Fenway Institute has published a brochure regarding the treatment of children. At its core is legal (parental or guardian) consent and this:

Demonstrated long-lasting, non-traditionalgender identity that results in significantdistress or gender dysphoria.

Persistent, documented gender dysphoria(this is part of what you will discuss with yourprovider)

Have another helping of baloney with green mold:

The philosophical problems highlight why this treatment protocol is misguidedindeed, why it violates sound norms of medical ethics. The purpose of medicine is to bring about human health and wholeness, human flourishing in the physical and psychological domains. Here health is understood not as the satisfaction of desires but as the well-functioning of the mind and body, where our various bodily systems achieve their endsthe circulatory system to circulate blood, the digestive system to digest nutrients, the respiratory system to absorb oxygen, etc.and where our thoughts and feelings achieve their ends of bringing us into contact with reality. Thus, any medical intervention intended to affirm someones false beliefs is inherently misguided. Affirming a falsehood via medical technology gets it wrong, right from the start.

Anderson was a music major turned philosopher. George is a lawyer. The care of children (which is at least what their title refers to) should be based on the best available science. Neither of these gentlemen have the training or experience to attack the science. They assert that gender-affirming care represents the satisfaction of desires as if it is gratuitous.

I have news for both of these people. A child in distress does not require a medical intervention to affirm their gender. They are inexorably drawn to do so without the assistance of anyone else.

Anderson's and George's polemic is full of preposterous hyperbole. The do just the opposite with gender dysphoria. They attempt to understate a child's understanding of their gender as some sort of false desire. Tell that to a kid in considerable distress or their parents.

The parents' response to this religious drivel will be one word: Bullshit. Shame on Ryan T. Anderson and Robert P. George for attempting to conform medical science to the teachings of the Catholic Church. They are not only manipulating the best science regarding treatment but they are falsely portraying what medical science knows about people (particularly children) with gender dysphoria.

Anderson and George go on to provide Five Points to Remember. I am going to give each a drivelectomy because Andergeorge are painfully verbose:

First, these procedures are entirely experimental. There is not a single long-term prospective study of the long-term consequences of blocking an otherwise physically healthy child from undergoing normal pubertal development. Indeed, the drugs being used to indefinitely delay normally timed puberty are not FDA-approved for this purpose and are being used off-label.

are these prepubescent children able to provide consent for the treatment? Giordano says that they can, so long as the clinician discusses all potential risks and benefits, as he or she must do with any experimental drug. Because this is the only therapy available for children with GID, it might be considered unethical to deny this treatment option.

It would be unethical to allow a patient to suffer through the distress of pubertal development when we have a way of preventing the distress it causes. Children and adolescents who suffer from gender identity disorder face significant physical, psychological, and social challenges, and receiving an inconsistent standard of medical care adds to those challenges.

[Dr.] Simona Giordano is Senior Lecturer in Bioethics at the School of Law, University of Manchester, UK. She is Programme Director of medical ethics teaching in undergraduate medical education in the School of Medicine, and also teaches for the Master and Postgraduate Diploma in Healthcare Ethics and Law. Simona is a member of the UK Register of Exercise Professionals, and qualified as an exercise instructor in 1999.

Second, parents are told that these procedures are fully reversible, but that is not true. Going off of puberty-blocking drugs, with the hope that development resumes, does nothing to reverse the delayed biologically appropriate development. You cant go back in time and reverse that delay.

Use of GnRH analogues doesn't cause permanent changes in an adolescent's body. Instead, it pauses puberty, providing time to determine if a child's gender identity is long lasting. It also gives children and their families time to think about or plan for the psychological, medical, developmental, social and legal issues ahead. If an adolescent child stops taking GnRH analogues, puberty will resume.

Third, many experts fear that these treatment protocols are self-fulfilling. Telling a little boy that he is a girl (or something else) or a girl that she is a boy (or something else), blocking his or her natural biological development into a man or a woman, and then flooding him or her with opposite-sex hormones will simply reinforce false beliefs.

Fourth, while the diagnosis that someone is of the opposite sex is medically and scientifically baseless, it is particularly outrageous when applied to children.

Fifth, and finally, not only is sex reassignment physically and metaphysically impossible, it doesnt even produce good psychosomatic results. So even if you disagreed with us about the philosophy of the body and the medical ethics of transitioning, you would still need to be concerned that an entirely experimental, self-fulfilling treatment protocol that is based on nonsensical diagnostic criteria doesnt even produce the desired outcomes of happiness and wholeness. Forty-one percent of all adults who identify as transgender attempt suicide at some point in their lives, and adults who have had sex reassignment surgery are nineteen times more likely than the general population to die by suicide.

I believe that the suicide statistics they quote is from a study assessing transgender surgery recipients going back 40 years. Surgery did not reduce minority stress. Anti-trans diatribes from religious lunatics increases minority stress. Nice job boys.

Included in point 5 is this bit of bullshit which I am paying attention to only to demonstrate how thoroughly dishonest these two are:

One more time, the title of this idiocy is: Physical Interventions on the Bodies of Children to Affirm their Gender Identity Violate Sound Medical Ethics and Should be Prohibited. What does any of that have to do with children? WPATH recommends that only adults are candidates for surgery.

I have not addressed two-thirds of this tirade. You can read it in full if you have some brain cells to spare. While they do not cite science to support their views Anderson and George provide numerous links to purchase Anderson's idiotic book on this subject which is nothing but an extension of the catechism.

I expect this kind of gibberish from Ryan T. Anderson. He is an over-educated religious zealot with the critical thinking skills of toilet tissue. I am disappointed in Robert P. George. In 2015 George had a meltdown over the ruling in Obergefell v. Hodges. He went so far as to promote nullification. Since then he mellowed on LGBTQ issues. George even came to the defense of Father James Martin who has questioned Church teachings regarding gay people.

Dr. George has considerable skill. I cannot imagine why he would lend his name and gravitas to something that is so profoundly dishonest.

The audience for this trash are like minded religious conservatives. What happens when one of them has a gender incongruent child? Are they going to risk a child's life to conform to the expectations of George and Anderson who torture medical science to conform to the teachings of the Catholic Church? Hopefully, they will consult with a secular psychiatrist for a proper evaluation.

Related content:

Read the rest here:
Ryan T. Anderson and Robert P. George Have Reached Conclusions About Trans Youth - The Slowly Boiled Frog

Recommendation and review posted by Bethany Smith

Founded in 1999, St. James Infirmary is a nonprofit organization based in San Francisco that provides free and confidential health care for sex workers. Recognizing the need for more compassionate care for sex workers, two associations, Call Off Your Old Tired Ethics (COYOTE) and the Exotic Dancers Alliance (EDA), founded the clinic in collaboration with the San Francisco Department of Public Health STD Control and Prevention Section. Its billed as an Occupational Health and Safety Clinic run by and for Sex Workers, past or present.

According to its website, St. James Infirmary provides upwards of 1,000 health care services to 300 unique patients, a syringe distribution and collection program, as well as education and training workshops that hundreds of sex workers attend, all in addition to basic primary care. The intention behind these classes is to dispense information about sexual health to take a more preventativemeasurewhen it comes public health and safety, rather than a punitive approach that results in jail time.

Named after the prolific sex-positive advocate Margo St. James, she, along with Carol Stuart and Jefferey Klausner, spearheaded activist collaboration, as profiled by a Jezebel interview. The clinic was immediately flooded with patientsdue to its welcoming and nonjudgmental atmosphere.

Speaking to Jezebel, Scarlett Paradise, a transgender woman, was given fundamental shelter and clothing at St. James. She said without the emotional support and sense of community St. James Infirmary offers, she wouldnt have survived.

The clinic is still running strong in San Francisco today, with a reported annual budget of $1 million dollars and moved its location to the Tenderloin District in San Francisco.It is expanding its services to counseling and mental health programs, hormone therapy, as well as partnering with OBGYN student residents from the University of California, San Francisco. St. James welcomes people of all sexual orientations, races, genders and backgrounds.

See original here:
The first health care clinic run "by sex workers for sex workers" is 20 years old - The Hill

Recommendation and review posted by Bethany Smith

CHICAGO--(BUSINESS WIRE)--Xeris Pharmaceuticals, Inc. (Nasdaq: XERS), a specialty pharmaceutical company leveraging its novel technology platforms to develop and commercialize ready-to-use injectable and infusible drug formulations, today announced positive topline results from the in-clinic stage of a Phase 2 study of its developmental ready-to-use (RTU) glucagon in patients who experience postprandial hypoglycemic episodes following bariatric surgery.

This is a Phase 2 prospective, randomized, placebo-controlled, double-blind study that comprises an in-clinic stage followed by a 12-week outpatient stage. Subjects are randomly assigned to receive RTU glucagon or placebo during two separate meal challenges in an in-clinic stage crossover design, and then enter a parallel design outpatient stage where they are assigned to an investigational product for 12 weeks. In this study, subjects self-administer a mini dose (300 g) of RTU glucagon or placebo when they experience hypoglycemia symptoms (e.g., anxiety, nausea, sweating, tremors, palpitations), and blood glucose response is measured after the study drug is self-administered. In situations where hypoglycemia (blood glucose 70 mg/dl) is present at mini-dosing or continues after treatment, oral glucose tabs are used in addition to the study drug. The in-clinic stage is now complete, and this study is currently ongoing in the outpatient stage, where both subjects and investigators remain blinded. For more information, visit http://www.clinicaltrials.gov Identifier: NCT03770637

Results from the in-clinic stage of this Phase 2 study demonstrate that most subjects experienced postprandial hypoglycemia within 90-120 minutes after finishing the meal. Of patients with a successful meal challenge, all subjects were also able to self-administer a mini dose of study drug, as directed, during the setting of declining blood glucose. A mini dose of RTU glucagon was adequate to restore or maintain normal blood glucose levels within 15 minutes of administration. This effect was maintained at 30 minutes, and hyperglycemia was not observed. The incidence of a follow-on episode of hypoglycemia (rebound hypoglycemia) requiring oral glucose for rescue was less with RTU glucagon compared to placebo. Treatment emergent adverse events with a mini dose of RTU glucagon were comparable to placebo, including negligible injection site reactions. Mini doses of RTU glucagon appear safe and well tolerated, and no serious adverse events occurred.

We are encouraged by the results of the in-clinic stage of our PBH study. The first half of this study is an important first step in demonstrating the utility of liquid, stable, ready-to-use glucagon in conditions beyond rescue for severe hypoglycemia and demonstrating safety and effectiveness in situations that require self-administration by the patient, said Paul R. Edick, Xeris Chairman and CEO. We believe the second half of the study, which is outside of the controlled in-clinic environment, will go further in establishing the safety profile of mini dosing RTU glucagon. That additional data will be available in the first half of 2020.

About Post-Bariatric Hypoglycemia (PBH)

Approximately 200,000 weight loss (bariatric) surgeries are performed annually in the United States. Hypoglycemia that occurs after bariatric and other forms of upper gastrointestinal surgery is a condition called post-bariatric hypoglycemia (PBH). It usually occurs >6 months to 8 years after surgery and is an uncommon and rarely reported metabolic complication that can be severe and disabling for some patients. Hypoglycemia episodes from PBH occur 1-3 hours after meals (postprandial hypoglycemia), often at a frequency of >10 times per month. Persistent or unrecognized hypoglycemia from PBH can progress to severe hypoglycemia (blood glucose <54 mg/dL) with symptoms such as loss of consciousness, seizures, coma, and even death. When postprandial hypoglycemia episodes in PBH occur, they can be difficult to acutely treat with oral carbohydrates alone, because an overcompensation with oral carbohydrates can frequently trigger a subsequent hypoglycemia episode (rebound hypoglycemia).

About Glucagon

Glucagon is a metabolic hormone secreted by the pancreas that raises blood glucose levels by causing the liver to rapidly convert glycogen (the stored form of glucose) into glucose, which is then released into the bloodstream. Glucagon and insulin are two critical hormones in a glycemic control system that keep blood glucose at the right level in healthy individuals. In people with diabetes who are dependent on insulin, this control system is disrupted, and insulin must be injected to avoid high levels of blood glucose (hyperglycemia). The opposite effect, or low blood glucose (hypoglycemia), is also prevalent in this population due to dysregulated glucagon secretion. Severe hypoglycemia is a serious condition and can lead to seizures, coma, potential brain injury and, if untreated, death.

Glucagon is the standard of care for treating severe hypoglycemia. According to the American Diabetes Association, glucagon should be prescribed for all individuals at increased risk of clinically significant hypoglycemia, defined as blood glucose <54 mg/dL (3.0 mmol/L). Leveraging XeriSol, one of Xeris two proprietary formulation technology platforms, Xeris has the potential to provide the first ready-to-use, room-temperature stable liquid glucagon for use by people with diabetes and other conditions to prevent or manage various forms of hypoglycemia and improve glucose control.

About Xeris Pharmaceuticals, Inc.

Xeris (Nasdaq: XERS) is a specialty pharmaceutical company delivering innovative solutions to simplify the experience of administering important therapies that people rely on every day around the world. With a novel technology platform that enables ready-to-use, room-temperature stable formulations of injectable and infusible therapies, the company is advancing a portfolio of solutions in various therapeutic categories, including its first commercial product, Gvoke. Its proprietary XeriSol and XeriJect formulation technologies have the potential to offer distinct advantages over conventional product formulations, including eliminating the need for reconstitution, enabling long-term, room-temperature stability, significantly reducing injection volume, and eliminating the requirement for intravenous (IV) infusion. With Xeris technology, new product formulations are designed to be easier to use by patients, caregivers, and health practitioners and help reduce costs for payers and the healthcare system.

Xeris is headquartered in Chicago, IL. For more information, visit http://www.xerispharma.com, or follow us on Twitter, LinkedIn or Instagram.

Forward-Looking Statements

Any statements in this press release about future expectations, plans and prospects for Xeris Pharmaceuticals, Inc., including statements regarding the acceptance of Gvoke in the marketplace, the market and therapeutic potential of its product candidates, expectations regarding clinical data, the timing or likelihood of regulatory approval and commercialization of its product candidates, the timing or likelihood of expansion into additional markets, expectations regarding the timing of the commercial launch of Gvoke HypoPen, the potential utility of its formulation platforms and other statements containing the words "will," "would," "continue," and similar expressions, constitute forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including, without limitation, the regulatory approval of its product candidates, its ability to market and sell its products, if approved, its reliance on a single source supplier for Gvoke HypoPen and other factors discussed in the "Risk Factors" section of the most recently filed Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission, as well as discussions of potential risks, uncertainties, and other important factors in Xeris subsequent filings with the Securities and Exchange Commission. Any forward-looking statements contained in this press release speak only as of the date hereof, and Xeris expressly disclaims any obligation to update any forward-looking statements, whether as a result of new information, future events or otherwise.

The Company intends to use the investor relations portion of its website as a means of disclosing material non-public information and for complying with disclosure obligations under Regulation FD.

Follow this link:
Xeris Pharmaceuticals Announces Positive Topline Results From the In-clinic Stage of the Phase 2 Study of Its Developmental Ready-to-use (RTU)...

Recommendation and review posted by Bethany Smith

Ash Lammin: Not everyone is going to understandSWNS

A 12-year-old who identifies as a girl is about to become one of the youngest children in Britain to take hormone blockers in her bid to change gender.

Ash Lammin, who was born a boy, changed her name from Ashton by deed poll aged eight. Her mother Terri, 43, who has seven other children, said: Although she was born male, from the moment she could speak Ash insisted she was a girl.

Ash, from Ramsgate, Kent, is about to start the transition from male to female at an NHS-run clinic by taking beta blockers that stop puberty.

She will decide whether she wants gender reassignment surgery when she is 18. If not she will come off the blockers and go through puberty a few years

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Ash Lammin, 12, to take hormone blockers | News - The Times

Recommendation and review posted by Bethany Smith

Someone cut you off on the highway and you had to swerve andnarrowly avoided a collision.

Cleveland Clinic is a non-profit academic medical center. Advertising on our site helps support our mission. We do not endorse non-Cleveland Clinic products or services.Policy

While out for a morning run, an angry dog jumps out onto your path and starts growling and barking at you.

In the second before you turned on the lights in your empty house,your coat rack looked like it was a person standing right next to you.

All three of these scenarios can trigger your bodys naturalfight or flight response, which is driven from your sympathetic nervous system.This response is your bodys reaction to danger and was designed to help yousurvive stressful and life-threating situations.

The fight or flight response, or stress response, is triggered by a release of hormones either prompting us to stay and fight or run away and flee, explains psychologist Carolyn Fisher, PhD. During the response, all bodily systems are working to keep us alive in what weve perceived as a dangerous situation.

Without you even telling it what to do, your body is assessing whats going on around you and determining your options on how you most likely could survive the event.

Heres what can happen during the stress response:

During the fight or flight response your body is trying to prioritize, so anything it doesnt need for immediate survival is placed on the back burner. This means that digestion, reproductive and growth hormone production and tissue repair are all temporarily halted. Instead, your body is using all its energy on the most crucial priorities and functions.

The stress response can be triggered in a single instant, but how quickly you calm down and return to your natural state is going to vary from person to person (and it will depend on what caused it). Typically it takes 20 to 30 minutes for your body to return to normal and to calm down.

Our fight or flight response was designed to help usthrough catastrophic circumstances, says Dr. Fisher. If you think about itfrom an evolution standpoint, it makes sense because we used to have a lot morelife-threatening emergencies.

Back in the caveman days, danger was all around us andthreats were constant. We didnt know where our next meal was coming from, wehad to brave the weather and we had to fight predators all around us. Arustling bush could be a lion or something else trying to kill you.

And so our ancestors developed the stress response to helpus survive.

Fortunately in todays word, real danger is few and farbetween, but that doesnt mean weve lost our ability to trigger the fight orflight response. It might happen while youre on an airplane thats experiencingturbulence or when someone jumps out at you from a dark room. And itll morethan likely be triggered if youre in a car accident, being robbed orexperiencing something else traumatic.

Where it gets tricky is when your body starts triggering thefight or flight response during non-threating situations like giving a bigpresentation, trying to make a deadline at work or merely thinking about a phobia, such as spiders or heights. Thesesituations arent truly dangerous, but theyve triggered our stress responseand our body is reacting to it as if it was.

In evolution, the stress response was designed to help us survive, but thats not always how it plays out in todays world, says Dr. Fisher. Our fight or flight response can now be activated from psychological or mental stress. For example, some individuals can activate it just thinking about work tomorrow.

Living in a prolonged state of high alert and stress (when there isnt any real reason for it) can be detrimental to your physical and mental health.

Your autonomic nervous system is a delicate balancing actbetween your sympathetic nervous system and your parasympathetic nervoussystem. Both networks involuntarily react to the environment around you.

Your sympathetic nervous system is responsible for how your body reacts to danger and is responsible for the fight or flight response. While your parasympathetic nervous system is responsible for maintaining homeostasis, which is your bodys built-in stability monitor. Think of it like a generator making sure everything from your body temperature to your water intake is functioning smoothly. Your parasympathetic nervous system makes sure things are balanced. It works to relax you and helps conserve and restore energy.

You need both systems to run properly.

Think of your sympathetic nervous system and yourparasympathetic nervous system like your cars gas and breaks, explains Dr.Fisher. You need to use both effectively for your car to run properly.

You need your sympathetic nervous system to keep you alive when true danger is detected and you need your parasympathetic nervous system to restore and relax you so that your body can run business as usual.

So if you find that your body is constantly reacting to every day stress with the fight or flight response it should be a warning sign that your sympathetic and parasympathetic systems arent working together in harmony.

Often times stressors that arent life threating dont havea clear on or off switch, says Dr. Fisher. Thats where we see some of thedetrimental effects of prolonged stress because its not going away. Its achronic stress to our immune system.

Work, bills, kids, your marriage, finances and health are some of the biggest non-life threatening stressors. How you interpret these things can affect your bodys reaction and can contribute to anxiety disorders.

Some people are having the fight or flight response whenthey go to work or see that their kid didnt clean up their room, says Dr.Fisher. It can vary from person to person in terms of the situations that cantrigger the stress response, but were finding that certain conditions orhealth states can be associated with this imbalance.

Some people who get in a car accident are too afraid to drive again or cant drive past the spot where the accident was because of fear and anxiety. It becomes a generalized fear response to a situation that isnt particularly dangerous anymore. This can also happen with work or strained relationships. The next thing you know, your fight or flight response is falsely activated, putting you in a state of chronic stress.

Dr. Fisher says stress management is critical to overallhealth. Its important to think big picture when you feel yourself starting toget worked up over something that you know is not a true threat or danger.

The fight or flight response is an important reaction that we all have and need, but its meant for true stress and danger. Everyone is going to have it in varying degrees for different reasons, but learning to slow down, be aware and conceptualize whats actually happening can help you regain control.

You need to get in touch with your individual physical,emotional and behavioral signs of stress, says Dr. Fisher. Maybe a migrainemeans youve had prolonged stress going on, so you need to tune into your bodyand whats going on before it gets to a crisis point.

If youre at the point where stress is impacting your quality of life talk to your doctor. Therapy, medication and stress management techniques can help you return to a more balanced state. Its not a quick fix and youll have to work on it daily, but you should be proactive about stress.

The fight or flight response has a clear purpose and function, but it shouldnt be activated over every day, non-threatening stressors like traffic, emails or bills. And if it is, the goal is to feel skilled at having an awareness when the response is activated, and to be able to bring yourself back to baseline.

Link:
What Happens to Your Body During the Fight or Flight Response? - Health Essentials from Cleveland Clinic

Recommendation and review posted by Bethany Smith

ONE of Britains youngest transgender children has started transitioning - after realising she was born in the wrong body aged just three.

Ash Lammin, 12, was born Ashton, but insisted that she was a girl as soon as she could speak at home in Ramsgate, Kent.

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Her mum Terri Lammin, 43, said that watching her daughter grow up confused and upset by her body was "heartbreaking".

She said: "Although she was born male, from the moment she could speak Ash insisted she was a girl.

"By age five, she was asking 'when is someone going to chop my winky off?' - and questioning why she had it at all."

Ash says that it has been difficult growing up as a trans girl, but says she feels that she is firmly on the right path.

She said: "The journey is long and it's still going, but I feel like the sense of victory is there through it all.

"I do feel accepted sometimes, but other times not.

"Not everyone is going to understand and people have to have their own opinions and I understand that. Some people might not like the idea of trans.

"I hope I inspire others but I just hope that love and acceptance comes through everything."

Now, aged almost 13, she is embarking on a lengthy journey to transition her gender from male to female at an NHS-run clinic - and is one of the youngest in the country to do so.

Ash - who changed her name by deed poll to Ashley when she was eight - will start by taking hormone blockers to halt the onset of puberty.

I do feel accepted sometimes, but other times not.

She has researched the process incessantly - and eventually wants a womb transplant so that she can be a mother when she's older.

Terri added: "I never thought it was a phase, Ash was just Ash.

"When she was three she said to me, 'I'm a boy because you gave me a boy's name - it's your fault.'

"I remember feeling horrible, because she blamed me. I personally thought maybe this was what an extremely camp gay man is like as a child."I'd never come across it before and I just went along with it. I just thought 'if he's happy, well that's the main thing.'"

But Terri, who has seven other children, said that life became much harder when Ash started at primary school.

She said: "I sent her to school in a boy's uniform. I felt awful, she didn't want to wear it and I was making her.

"The school were great. The headmaster at the time said 'if you think it's going to make life easier then bring Ash in a girl's uniform', so I did.

"I was in a right state. I thought 'everybody is going to think I'm weird' - but Ash loved it, she found it easy.

"Before, when I was taking her into school, she was biting me and kicking me, she didn't want to go in.

Although she was born male, from the moment she could speak Ash insisted she was a girl

"As soon as she put the girl's uniform on, she wanted to go every day."

Despite the school's willingness to help and the kindness of Ash's classmates, Terri says that other parents were very difficult - leaving her out of social events and complaining that Ash was using the girls' toilets.

She added: "When Ash was Ashton, she was invited to all the kids' parties, even though she used to turn up in a princess dress.

"The parents didn't mind then. But as soon as I let her be Ashley all the time, for a whole year she didn't get invited to one party.

"The kids were fine; it's not the children, kids play with anybody. It's not until an adult comes in and says you shouldn't do that then it changes."

When Ash turned 11 and went to secondary school, she became a target for bullies who would throw things at her on the bus and shout tranny at her.

Her mum was forced Terri to take her out of the school after just one term.

She is now being home-schooled, and Terri is calling for better education within schools to teach children about transgender people.

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View original post here:
One of Britains youngest transgender kids, 12, transitions after realising she was in wrong body aged 3 - The Sun

Recommendation and review posted by Bethany Smith

Its been just over a year since the dramatic announcement of the worlds first genome-edited babies using CRISPR technology. Since then, to the chagrin of some and the relief of others, there have been no more such announcements. This is due, in no small part, to discreet actions taken by the Peoples Republic of China, the World Health Organization (WHO) and the Russian Federation.

Read more: What is CRISPR gene editing, and how does it work?

In late November 2018, He Jiankui, a Chinese biophysicist, confirmed hed created genetically modified twins in an effort to provide the children with resistance to HIV. A few days later, he presented some of his work at the Second International Summit on Genome Editing in Hong Kong. At this meeting, He mentioned another ongoing pregnancy involving the use of a genetically modified embryo. To this day, we do not know the outcome of this pregnancy.

What we do know is that Chinas Ministry of Science and Technology condemned Hes actions and shortly thereafter, Chinas National Health Commission drafted new regulations on the clinical use of emerging biomedical technologies, including human genome editing. The final text of the Administrative Regulations for the Clinical Application of New Biomedical Technologies is not yet available and it is not known when these regulations will come into effect.

Based on the draft text open to public comment, research of the type conducted by He would require approval from Chinas highest administrative authority.

In the wake of Hes controversial experiment, the WHO convened a multi-disciplinary Expert Advisory Committee on Human Genome Editing to examine the scientific, ethical, social and legal challenges associated with human genome editing (both somatic and germ cell).

Specifically, the committee was tasked by the director general, Tedros Adhanom Ghebreyesus, to advise and make recommendations on appropriate governance mechanisms. The committee (of which I am a member) met for the first time in March 2019.

In June 2019, Russian molecular biologist Denis Rebrikov announced his plans to follow in Hes footsteps. Rebrikov would genetically modify early-stage human embryos in his lab and use those embryos to initiate a pregnancy that hopefully would result in the birth of healthy HIV-resistant offspring. Unlike He, however, Rebrikov planned to involve HIV-infected women in his research in an effort to address the risk of transmission of the virus in utero from the pregnant woman to her fetus. (Hes research involved HIV infected men.)

In response, on advice from the WHO Expert Advisory Committee, the WHO director general issued a statement calling on regulatory and ethics authorities in all countries to refrain from approving research on heritable human genome editing until its ethical and social implications had been properly considered.

Read more: Opening Pandora's Box: Gene editing and its consequences

Undeterred by the WHO announcement, in September and October 2019 Rebrikov, confirmed his intention to apply for permission to proceed with heritable human genome editing, but with a different focus. Though it was initially reported that Rebrikov felt a sense of urgency to help women with HIV, he was unable to find HIV-positive women who did not respond to standard anti-HIV drugs and who wanted to get pregnant to participate in his research.

So, instead of modifying the CCR5 gene which would provide future offspring with resistance to HIV, Rebrikov planned to modify the GJB2 gene to correct a mutation that causes a type of hereditary deafness. According to Rebrikov, there were several couples interested in participating in this research.

Meanwhile, the Russian government issued a statement making it clear that Rebrikov would not get regulatory approval for the proposed research.

In October 2019, the Ministry of Health of the Russian Federation affirmed that the use of heritable genome editing was premature. Further, the ministry officially endorsed the WHO position that it would be irresponsible and unacceptable to use genome-edited embryos to initiate human pregnancies.

Finally and most importantly the Ministry of Health explicitly stated that the WHO position, supported by the Russian Federation, should be decisive in the formation of country policies in this area.

This strong statement by the Ministry of Health of the Russian Federation is reassuring. It sets an important example for regulatory authorities around the world who support the WHOs efforts to develop effective governance instruments to deter and prevent irresponsible and unacceptable uses of genome editing of embryos to initiate human pregnancies.

In the last lines of my new book Altered Inheritance: CRISPR and the Ethics of Human Genome Editing I write:

As a direct consequence of increasingly audacious moves by some scientists to engineer future generations, important decisions must now be made decisions that will set a new course for science, society, and humanity. May these decisions be inclusive and consensual. May they be characterized by wisdom and benevolence. And, may we never lose sight of our responsibilities to us all.

Collectively, all of us (experts and non-experts) have a responsibility to make the best use of emerging technologies to improve the health and well-being of all people everywhere. This can only be achieved through collaborative effort on a global scale.

We need time to carefully consider the kind of world we want to live in and how human genome editing technology might or might not help us build that world. We cant do this work properly if scientists brashly go about the business of making genome-edited babies.

[ Youre smart and curious about the world. So are The Conversations authors and editors. You can read us daily by subscribing to our newsletter. ]

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A year after the first CRISPR babies, stricter regulations are now in place - The Conversation CA

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University of Otago researchers have discovered how viruses that specifically kill bacteria can outwit bacteria by hiding from their defenses, findings which are important for the development of new antimicrobials based on viruses and provide a significant advance in biological knowledge.

Lead researcher Professor Peter Fineran explains that the rise in multi-drug resistant bacteria is leading to the development of alternative therapeutics, including viruses that specifically kill bacteria, called bacteriophages, often referred to as phages. However, bacteria can become resistant to phages.

This is professor Peter Fineran. Credit: University of Otago

Phages are the most abundant biological entities on the planet and are important for global ecosystems, but they can also be used to kill bacterial pathogens. To defend themselves from the phage invasion, bacteria have developed CRISPR-Cas defense systems immune systems within the bacteria. But the phages have come up with many ways to avoid these bacterial defenses.

In the study published December 9, 2019, in Nature Microbiology, the team at the University of Otago discovered a widespread method used by phages to hide from bacterial defenses. They discovered a jumbo phage which, as the name suggests, is very big, with hundreds of genes. This phage is not recognized by CRISPR-Cas defenses that would normally cut up the genetic DNA instructions to make many new phages.

Ph.D. student in the Department of Microbiology and Immunology and first author of the study, Lucia Malone says it made the researchers question how this phage escapes recognition.

We had molecular and genetic evidence for what was happening, but we really needed to see directly inside these tiny bacteria, which if 100 lined up side-by-side would be the width of a human hair, Ms. Malone says.

This is first author of the study, PhD student Lucia Malone. Credit: University of Otago

This was made possible using a new spinning disk confocal microscope for high-resolution imaging of live cells the only one with this capability in New Zealand that was recently set up by Dr. Laura Gumy, a new group leader at the University of Otago.

When phages infected the bacteria, we could see their DNA was encased by a physical shield and hidden from the CRISPR-Cas defence systems that couldnt gain access, Dr. Gumy explains.

However, bacteria have another trick up their sleeve. To take over the host, the phages must produce RNA messages that leave this protective compartment. This is the Achilles heel of these phages and can be destroyed by a special group of CRISPR-Cas defenses that recognize RNA messages, Ms Malone says.

Dr. Fineran explains the study broadens the knowledge of intricate phage-host interactions and demonstrates that jumbo phages are less susceptible to bacterial defense systems than some other phages.

From a biological perspective, our results provide exciting new insights into how phages evade bacterial defense systems.

This is important because the rise of the multi-drug resistant bacteria is an issue of global concern, which has led to a renewed interest in using phages as anti-bacterials and jumbo phages may provide excellent therapeutics.

Reference: A jumbo phage that forms a nucleus-like structure evades CRISPRCas DNA targeting but is vulnerable to type III RNA-based immunity by Lucia M. Malone, Suzanne L. Warring, Simon A. Jackson, Carolin Warnecke, Paul P. Gardner, Laura F. Gumy and Peter C. Fineran, 9 December 2019, Nature Microbiology.DOI: 10.1038/s41564-019-0612-5

Link:
New Viral Strategy to Escape Detection Discovered by Researchers - SciTechDaily

Recommendation and review posted by Bethany Smith

The Global CRISPR Genome Editing Market report study includes an elaborative summary of the CRISPR Genome Editing market that provides in-depth knowledge of various different segmentations. CRISPR Genome Editing Market Research Report presents a detailed analysis based on the thorough research of the overall market, particularly on questions that border on the market size, growth scenario, potential opportunities, operation landscape, trend analysis, and competitive analysis of CRISPR Genome Editing Market. The information includes the company profile, annual turnover, the types of products and services they provide, income generation, which provide direction to businesses to take important steps. CRISPR Genome Editing delivers pin point analysis of varying competition dynamics and keeps ahead of CRISPR Genome Editing competitors such as Editas Medicine, CRISPR Therapeutics, Horizon Discovery, Sigma-Aldrich, Genscript, Sangamo Biosciences, Lonza Group, Integrated DNA Technologies, New England Biolabs, Origene Technologies, Transposagen Biopharmaceuticals, Thermo Fisher Scientific, Caribou Biosciences, Precision Biosciences, Cellectis, Intellia Therapeutics.

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The main objective of the CRISPR Genome Editing report is to guide the user to understand the CRISPR Genome Editing market in terms of its definition, classification, CRISPR Genome Editing market potential, latest trends, and the challenges that the CRISPR Genome Editing market is facing. In-depth researches and CRISPR Genome Editing studies were done while preparing the CRISPR Genome Editing report. The CRISPR Genome Editing readers will find this report very beneficial in understanding the CRISPR Genome Editing market in detailed. The aspects and information are represented in the CRISPR Genome Editing report using figures, bar-graphs, pie diagrams, and other visual representations. This intensifies the CRISPR Genome Editing pictorial representation and also helps in getting the CRISPR Genome Editing industry facts much better.

.This research report consists of the worlds crucial region market share, size (volume), trends including the product profit, price, Value, production, capacity, capability utilization, supply, and demand and industry growth rate.

Geographically this report covers all the major manufacturers from India, China, the USA, the UK, and Japan. The present, past and forecast overview of the CRISPR Genome Editing market is represented in this report.

The Study is segmented by following Product Type, Genetic Engineering, Gene Library, Human Stem Cells, Others

Major applications/end-users industry are as follows Biotechnology Companies, Pharmaceutical Companies, Others

CRISPR Genome Editing Market Report Highlights:

1)The report provides a detailed analysis of current and future market trends to identify the investment opportunities2) In-depth company profiles of key players and upcoming prominent players3) Global CRISPR Genome Editing Market Trends (Drivers, Constraints, Opportunities, Threats, Challenges, Investment Opportunities, and recommendations)4) Strategic recommendations in key business segments based on the market estimations5) To get the research methodologies those are being collected by CRISPR Genome Editing driving individual organizations.

Research Parameter/ Research Methodology

Primary Research:

The primary sources involve the industry experts from the Global CRISPR Genome Editing industry including the management organizations, processing organizations, analytics service providers of the industrys value chain. All primary sources were interviewed to gather and authenticate qualitative & quantitative information and determine future prospects.

In the extensive primary research process undertaken for this study, the primary sources industry experts such as CEOs, vice presidents, marketing director, technology & innovation directors, founders and related key executives from various key companies and organizations in the Global CRISPR Genome Editing in the industry have been interviewed to obtain and verify both qualitative and quantitative aspects of this research study.

Secondary Research:

In Secondary research crucial information about the industry value chain, the total pool of key players, and application areas. It also assisted in market segmentation according to industry trends to the bottom-most level, geographical markets and key developments from both market and technology oriented perspectives.

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Global CRISPR Genome Editing Market 2019 by Company, Regions, Type and Application, Forecast to 2025 - The Market-News 24

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Clustered Regularly Interspaced Short Palindromic Repeats(CRISPR) Technology Market report offers detailed analysis and a five-year forecast for the global Clustered Regularly Interspaced Short Palindromic Repeats(CRISPR) Technology industry. Clustered Regularly Interspaced Short Palindromic Repeats(CRISPR) Technology market report delivers the insights which will shape your strategic planning as you estimate geographic, product or service expansion within the Clustered Regularly Interspaced Short Palindromic Repeats(CRISPR) Technology industry.. Global Clustered Regularly Interspaced Short Palindromic Repeats(CRISPR) Technology Market Report is a professional and comprehensive research report on the worlds major regional market conditions, focusing on the main regions (North America, Europe and Asia-Pacific) and the main countries (United States, Germany, United Kingdom, Japan, South Korea and China).

In this report, the global Clustered Regularly Interspaced Short Palindromic Repeats(CRISPR) Technology market is valued at USD XX million in 2019 and is projected to reach USD XX million by the end of 2024, growing at a CAGR of XX% during the period 2019 to 2024.

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Clustered Regularly Interspaced Short Palindromic Repeats(CRISPR) Technology Market with Future Prospects, Key Player SWOT Analysis and Forecast To...

Recommendation and review posted by Bethany Smith

Hormone deficiency is treated by replacing the deficient hormones. The goals of treatment are to improve symptoms (see Table 2) and to replace the deficient hormone or hormones at a level that is as close to physiologically correct (mother nature) as possible. However, one rule of hormone replacement is that no one dose will suit every patient. Thus, when hormone replacement therapy is prescribed, the patient will need to be seen regularly after starting treatment to assess the effect. It often takes time and repeated dose changes to find the optimal dose for each patient. Typically, once the optimal dose is determined, the dose remains adequate for long-term treatment unless other medications are added or the patients condition changes in a way that alters the blood levels (e.g., introduction of GH therapy may require an increase in cortisol replacement, whereas pregnancy may require an increase in the dose of thyroid hormone).

Cortisol: On average, cortisol replacement therapy consists of giving approximately 15 mg of cortisol daily in divided doses. Approximately 2/3 of the dose is given in the morning and 1/3 in the late afternoon or evening. Excess cortisol can cause side effects (see the section on risks below), so it is best to use cortisol replacement in doses that are adequate but not too high.

Some endocrinologists prescribe prednisone instead of cortisol, and the dose of prednisone can be given once or twice a day. Patients with cortisol deficiency must always remember that during periods of stress their bodies may not be able to produce the increased level of cortisol needed. Therefore, patients should always carry a medical or steroid alert card or wear a medical alert bracelet or necklace to inform physicians that they are taking chronic steroid therapy. If patients have multiple pituitary hormone deficiencies, cortisol should always be the first hormone replaced as medications like thyroid hormone or GH can increase the bodys need for cortisol.

Thyroid hormone:Levothyroxine given daily is the therapy for thyroid hormone deficiency.

Sex-related hormones:Women: Premenopausal women who have no menstrual cycles as a result of pituitary disease (secondary hypogonadism) should receive replacement therapy with estrogen and progesterone. Estrogen can be given orally, by patch or by gel. Progesterone equivalent is only required in woman who have an intact uterus. Women who have undergone a hysterectomy can be treated with estrogen alone.

Men: In testosterone-deficient men, testosterone is given by patch, gel or injection either daily (patch or gel) or every 2-4 weeks by intramuscular injection.

GH therapy:GH prescribing practices vary depending upon local customs, national guidelines and insurance coverage. It is important to do tests to prove that patients are indeed GH deficient. Human GH is administered by daily injection. Most pituitary endocrinologists start at relatively low doses to avoid side effects and increase as needed.

DI therapy:Desmopressin is usually given in tablet or spray form (nasal tube or nasal spray). Hospitalized patients may be given desmopressin by injection.

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How Hypopituitarism is Treated | Learn More About ...

Recommendation and review posted by Bethany Smith

On the heels of an FDA speedy review for Keytrudas potential use in non-muscle invasive bladder cancer (NMIBC), its close rival, a gene therapy by Ferring Pharmaceuticals spinout FerGene, has posted late-stage data. By the looks of it, the two drugs are up for a fight.

Among patients with high-risk NMIBC superficial disease thats unresponsive to standard-of-care Bacillus Calmette-Gurin (BCG), nadofaragene firadenovec eliminated tumors in 53%, or 55 of 103 patients,at month three in a phase 3 study, FerGene unveiled Thursday at the Society of Urologic Oncology meeting.

By comparison, in Keytrudas own registrational trial on the same target patient population, the Merck & Co. PD-1 completely cleared tumors in 41.2%, or 42 of 102 patients, after three months, according to an update at the European Society for Medical Oncology annual meeting in September.

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The length of time responses lasted appeared similar between the two drugs in their separate studies. For Keytruda, 24 patients (23.5%) continued to show no signs of disease after a year. As for nadofaragene firadenovec, 24.3%, or 25 patients, were still tumor-free at month 12.

In terms of safety, Keytruda recorded Grade 3/4 side effects in12.7% of patients, while FerGene said there were no Grade 4/5 events in its study.

We are pleased with these Phase 3 data results, including the complete response rates and favorable safety profile seen with nadofaragene firadenovec, Nigel Parker, scientific founder of FKD Therapies, said in a statement. The data have also helped FKD'snew drug application earn an FDA priority review.

RELATED:Merck's Keytruda is bound for new bladder cancer territory. But can it hold up against gene therapy?

Ferring recently gained commercial rights to the gene therapy from FKD, and, with $400 million in help from Blackstone Life Sciences, spun it into FerGene. Interestingly, it was Merck that licensed the drugout to FKD in the first place in 2011 in return for an equity stake in the then-newly formed Finnish company.

Priority reviews in hand, the two companies could be looking at FDA approvals soon. The burning question is, how does FerGene plan to price a gene therapy, which belongs to a class of drug thats notoriously costly? In a statement sentto FiercePharma, Ferring said it's too early to discuss pricing, that its top priority is still to get nadofaragene firadenovec approved andinvest into R&Dto study the product in more indications.

Keytruda is meant to be given ata fixed dose every three weeks. Nadofaragene firadenovec, which uses an adenovirus vector to deliver the gene interferon alfa-2b to stimulate an innate immune response to fight cancer, is administered into the bladder every three months.

Merck does have an upper hand against FerGene. The Big Pharma has been the sole supplier of BCG in the U.S. and several other key markets globally for several years now. So, it could offer BCG and Keytruda as a one-two punch for NMIBC, similar to the wayBayer is billing Nexavar and Stivarga as a part of the same continuumin first- and second-line liver cancer.

RELATED:Merck limits orders for bladder cancer drug as demand outstrips supply

There are other players eyeing the same patient population. Sesen Bio has Vicinium, an antibody-drug conjugate that targets epithelial cell adhesion molecule antigens on the surface of tumor cells to deliver a toxin payload. In its own phase 3 trial dubbed Vista also in high-risk, BCG-unresponsive NMIBC, Vicinium eradicated tumors in 40% of 89 patients at month three, according to an update the company provided in August. However, its response seems to wane over time more quickly than its rivals', as only 17% of patients showed no signs of tumor activity after 12 months.

The Cambridge, Massachusetts-based biotech recently held two meetings with the FDA and confirmed a submission process, including the design for a post-marketing confirmatory trial. It would enroll BCG-refractory patients who, because of supply constraints, haven't received an optimal BCG dose, which the company said represents a broader patientpopulation in light of anongoing shortage.

Sesen now expects to submit a biologics license application under rolling review by year-end with potential approval in 2020.

As for its pricing, during a presentation at the H.C. Wainwright investor conference in September, Sesens president and CEO Thomas Cannell pointed out that PD-1/L1s would cost about $150,000 to $200,000 per patient per year in NMIBC.

Weve done two rounds of market research with payers, and they think thats reasonable, he said. They think at those levels, there will probably be minimal prior authorization or step edits in terms of restricting a treatments use.

Assuming an official launch in 2021, Jefferies analysts, in a Nov. 12 note to clients, pegged $167.5 million for Viciniums U.S. sales in 2024. Before the priority designation, SVB Leerinks Daina Graybosch predicted a Keytruda launch in NMIBC in 2022 and forecastU.S. sales of $250 million in the indication for the Merck PD-1 inhibitor in 2025.

Editor's Note: The story has been updated with a statement from Ferring Pharma.

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Watch out, Keytruda. Ferring's bladder cancer gene therapy rival has new dataand they look competitive - FiercePharma

Recommendation and review posted by Bethany Smith

Understanding Real-World Evidence to Advance Patient-Centric Innovation in Bleeding Disorders

Real-world evidence from studies across many of Takedas portfolio of treatments for hemophilia demonstrate the cost savings and patient benefits resulting from ongoing personalized treatment. However, in von Willebrand disease, real-world evidence highlights the ongoing unmet clinical need for personalization, as it may enable improved treatment outcomes. Insights presented at ASH include:

Real-world evidence plays a crucial role in understanding patterns of care that happen in day-to-day medical practice outside of rigorous clinical studies, said Jonathan Roberts, MD, Associate Medical Director, Bleeding & Clotting Disorders Institute, Peoria, Ill. The U.S. medical claims data show improvement is needed around management of von Willebrand disease in children and adolescents to optimize treatment and reduce the amount of bleeding episodes following diagnosis.

Preclinical Scientific Studies Address Challenges of Current AAV Gene Therapies Takeda also presented data from preclinical scientific studies regarding certain known limitations of AAV gene therapies. These studies will inform Takeda's approach to its own investigational AAV gene therapy programs; TAK-754, an investigational AAV gene therapy for hemophilia A is currently in Phase 1 clinical study, soon to be followed by other potential gene therapies including TAK-748, an investigational gene therapy for hemophilia B.

The treatment goal of gene therapy for hemophilia is to provide sustained therapeutic levels of endogenous clotting factor over multiple years. Hemophilia gene therapies have the potential to provide prolonged, high-level expression of factor, and limit the need for frequent factor infusion.1,2 To deliver gene therapy to a patient, a normal copy of a missing gene is packaged into a delivery vehicle, called a vector.3 Recombinant AAV, particularly those delivered by AAV5 and AAV8 capsid serotypes, serve as the vector in most of the ongoing hemophilia studies.3 The vector delivers the functional gene into a patients liver cells, which can then properly produce blood-clotting proteins. 4,5 However, patients pre-existing immunity to AAV8 capsid, and other AAV serotypes, can impact the safety and efficacy of these therapies.3

To better understand the prevalence of pre-existing immunity against commonly used AAV2, AAV5 and AAV8 capsid in adult patients with hemophilia, Takeda conducted an international prospective and ongoing epidemiological study, Co-Prevalence of Pre-Existing Immunity to Different Serotypes of Adeno-Associated Virus (AAV) in Adults with Hemophilia, (abstract 3349) that found 50% of patients with hemophilia have neutralizing antibodies to AAV2, AAV5 or AAV8 capsid with 40% demonstrating co-prevalence to all three evaluated serotypes. As a result, these patients are not likely to respond to gene therapies based on AAV vectors.3

As we continue to advance our hemophilia A and hemophilia B investigational gene therapy programs, Takeda is also investigating approaches to overcome the challenges of current AAV gene therapies that could potentially be applied to hemophilia and other rare monogenic diseases, said Dan Curran, M.D., Head, Rare Diseases Therapeutic Area Unit at Takeda. Developing new gene therapy approaches including those capable of treating pre-existing immunity to AAV, enabling re-dosing, lowering doses, enhancing biodistribution and developing alternative gene delivery vehicles are critical to one day providing functional cures to patients.

The poster AAV8-Specific Immune Adsorption Column: A Treatment Option for Patients with Pre-Existing Anti-AAV8 Neutralizing Antibodies, (abstract 5922) reported pre-clinical data on one potential approach to overcoming pre-existing AAV immunity.6 In the study, an AAV8-specific immune adsorption column (IAC) was used to mimic the processing of patients plasma in an in vitro setting by applying different treatment cycles to plasma reservoirs which shows anti-AAV8 titers could be depleted.6 Insights from this study will be applied to Takedas research to determine if an IAC could enable the administration of AAV8 gene therapies to patients with pre-existing immunity and potentially facilitate the re-administration of gene therapy.6

ADYNOVATE Professional Important Information

ADYNOVATE [Antihemophilic Factor (Recombinant), PEGylated] Important Information

Indications and Limitation of Use ADYNOVATE is a human antihemophilic factor indicated in children and adults with hemophilia A (congenital factor VIII deficiency) for:

ADYNOVATE is not indicated for the treatment of von Willebrand disease.

DETAILED IMPORTANT RISK INFORMATION

CONTRAINDICATIONS Prior anaphylactic reaction to ADYNOVATE, to the parent molecule (ADVATE [Antihemophilic Factor (Recombinant)]), mouse or hamster protein, or excipients of ADYNOVATE (e.g. Tris, mannitol, trehalose, glutathione, and/or polysorbate 80).

WARNINGS & PRECAUTIONSHypersensitivity Reactions Hypersensitivity reactions are possible with ADYNOVATE. Allergic-type hypersensitivity reactions, including anaphylaxis, have been reported with other recombinant antihemophilic factor VIII products, including the parent molecule, ADVATE. Early signs of hypersensitivity reactions that can progress to anaphylaxis may include angioedema, chest tightness, dyspnea, wheezing, urticaria, and pruritus. Immediately discontinue administration and initiate appropriate treatment if hypersensitivity reactions occur.

Neutralizing Antibodies Formation of neutralizing antibodies (inhibitors) to factor VIII can occur following administration of ADYNOVATE. Monitor patients regularly for the development of factor VIII inhibitors by appropriate clinical observations and laboratory tests. Perform an assay that measures factor VIII inhibitor concentration if the plasma factor VIII level fails to increase as expected, or if bleeding is not controlled with expected dose.

ADVERSE REACTIONS The most common adverse reactions (1% of subjects) reported in the clinical studies were headache and nausea.

Click here for Full Prescribing Information https://www.shirecontent.com/PI/PDFs/ADYNOVATE_USA_ENG.pdf

FEIBA [Anti-Inhibitor Coagulant Complex] Indications and Detailed Important Risk Information

Indications for FEIBA

FEIBA is an Anti-Inhibitor Coagulant Complex indicated for use in hemophilia A and B patients with inhibitors for:

FEIBA is not indicated for the treatment of bleeding episodes resulting from coagulation factor deficiencies in the absence of inhibitors to coagulation factor VIII or coagulation factor IX.

Detailed Important Risk Information for FEIBA

WARNING: EMBOLIC AND THROMBOTIC EVENTS

CONTRAINDICATIONS

FEIBA is contraindicated in patients with:

WARNINGS AND PRECAUTIONS

Thromboembolic events (including venous thrombosis, pulmonary embolism, myocardial infarction, and stroke) can occur, particularly following the administration of high doses (>200 units/kg/day) and/or in patients with thrombotic risk factors.

Patients with DIC, advanced atherosclerotic disease, crush injury, septicemia, or concomitant treatment with recombinant factor VIIa have an increased risk of developing thrombotic events due to circulating tissue factor or predisposing coagulopathy. Potential benefit of treatment should be weighed against potential risk of these thromboembolic events.

Infusion should not exceed a single dose of 100 units/kg and daily doses of 200 units/kg. Maximum injection or infusion rate must not exceed 2 units/kg/minute. Monitor patients receiving >100 units/kg for the development of DIC, acute coronary ischemia and signs and symptoms of other thromboembolic events. If clinical signs or symptoms occur, such as chest pain or pressure, shortness of breath, altered consciousness, vision, or speech, limb or abdomen swelling and/or pain, discontinue FEIBA and initiate appropriate diagnostic and therapeutic measures.

Safety and efficacy of FEIBA for breakthrough bleeding in patients receiving emicizumab has not been established. Cases of thrombotic microangiopathy (TMA) were reported in a clinical trial where subjects received FEIBA as part of a treatment regimen for breakthrough bleeding following emicizumab treatment. Consider the benefits and risks with FEIBA if considered required for patients receiving emicizumab prophylaxis. If treatment with FEIBA is required for patients receiving emicizumab, the hemophilia treating physician should closely monitor for signs and symptoms of TMA. In FEIBA clinical studies TMA has not been reported.

Hypersensitivity and allergic reactions, including severe anaphylactoid reactions, can occur. Symptoms include urticaria, angioedema, gastrointestinal manifestations, bronchospasm, and hypotension. Reactions can be severe and systemic (e.g., anaphylaxis with urticaria and angioedema, bronchospasm, and circulatory shock). Other infusion reactions, such as chills, pyrexia, and hypertension have also been reported. If signs and symptoms of severe allergic reactions occur, immediately discontinue FEIBA and provide appropriate supportive care.

Because FEIBA is made from human plasma it may carry a risk of transmitting infectious agents, e.g., viruses, the variant Creutzfeldt-Jakob disease (vCJD) agent and, theoretically, the Creutzfeldt-Jakob disease (CJD) agent.

FEIBA contains blood group isohemagglutinins (anti-A and anti-B). Passive transmission of antibodies to erythrocyte antigens, e.g., A, B, D, may interfere with some serological tests for red cell antibodies, such as antiglobulin test (Coombs test).

ADVERSE REACTIONS

Most frequently reported adverse reactions observed in >5% of subjects in the prophylaxis trial were anemia, diarrhea, hemarthrosis, hepatitis B surface antibody positive, nausea, and vomiting.

Serious adverse reactions seen are hypersensitivity reactions and thromboembolic events, including stroke, pulmonary embolism and deep vein thrombosis.

DRUG INTERACTIONS

Consider possibility of thrombotic events when systemic antifibrinolytics such as tranexamic acid and aminocaproic acid are used with FEIBA. No adequate and well-controlled studies of combined or sequential use of FEIBA and recombinant factor VIIa, antifibrinolytics, or emicizumab, have been conducted. Use of antifibrinolytics within approximately 6 to 12 hours after FEIBA is not recommended.

Clinical experience from an emicizumab clinical trial suggests that a potential drug interaction may exist with emicizumab.

Please see FEIBA full Prescribing Information, including BOXED WARNING on Embolic and Thrombotic Events

About Hemophilia Hemophilia is a challenging chronic disease that causes longer-than-normal bleeding due to absent or deficient clotting factor in the blood.7 Hemophilia A is more common than hemophilia B;7 hemophilia A affects about 158,225 people, whereas hemophilia B affects about 31,247 people worldwide.8

People with hemophilia, working closely with their healthcare professionals, can live healthy lives with proper care and adequate treatment.7 Treatment regimens typically include on-demand and/or regular prophylactic infusions of factor replacement therapy to control or prevent the risk of bleeding.7,8

About Takeda Hematology Following its recent acquisition of Shire, Takeda is a leader in hemophilia with the longest heritage and market-leading portfolio, backed by established safety and efficacy profiles with decades of real-world experience. We have 70+ years driving innovation for patients9 and a broad portfolio of 11 products across multiple bleeding disorders.10 Our experience as leaders in hematology means we are well prepared to meet todays needs as we pursue future developments in the care of bleeding disorders. Together with the hematology community, we are raising expectations for the future, including earlier diagnosis, earlier and full protection against bleeds, and more personalized patient care.

About Takeda Pharmaceutical Company Takeda Pharmaceutical Company Limited (TSE:4502/NYSE:TAK) is a global, values-based, R&D-driven biopharmaceutical leader headquartered in Japan, committed to bringing Better Health and a Brighter Future to patients by translating science into highly-innovative medicines. Takeda focuses its R&D efforts on four therapeutic areas: Oncology, Gastroenterology (GI), Neuroscience and Rare Diseases. We also make targeted R&D investments in Plasma-Derived Therapies and Vaccines. We are focusing on developing highly innovative medicines that contribute to making a difference in people's lives by advancing the frontier of new treatment options and leveraging our enhanced collaborative R&D engine and capabilities to create a robust, modality-diverse pipeline. Our employees are committed to improving quality of life for patients and to working with our partners in health care in approximately 80 countries and regions.

For more information, visit https://www.takeda.com.

References 1. National Hemophilia Foundation. Future Therapies. Accessible at: https://www.hemophilia.org/Bleeding-Disorders/Future-Therapies. Accessed: November 2019.2. Wong, T. & Recht, M. Drugs (2011) 71: 305.3. Rajavel, K et al. Co-Prevalence of Pre-Existing Immunity to Different Serotypes of Adeno-Associated Virus (AAV) in Adults with Hemophilia. Data on File.4. Doshi BS, Arruda VR. Gene Therapy for Hemophilia: What Does the Future Hold? Therapeutic Advances in Hematology. 2018; 9(9):273-293.5. Pipe, SW. Gene therapy for hemophilia. Pediatric Blood Cancer. 2018; 65(2): e26865.6. Kruzik, A et al. AAV8-specific immune adsorption column: A treatment option for patients with pre- existing anti-AAV8 neutralizing antibodies. Data on File.7. World Federation of Hemophilia. What is hemophilia? World Federation of Hemophilia website. http://www.wfh.org/en/page.aspx?pid=646. Last Accessed April 2019.8. World Federation of Hemophilia. Report on the Annual Global Survey 2017. World Federation of Hemophilia website. http://www1.wfh.org/publications/files/pdf-1714. pdf Last Accessed April 2019.9.World Federation of Hemophilia. About Bleeding Disorders: Treatment. World Federation of Hemophilia website. https://www.wfh.org/en/page.aspx?pid=642. Last Accessed April 2019.10. Shire Website. Product List. Website: https://www.shire.com/products/product-list?t=. Last Accessed June 2019.

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This press release and any materials distributed in connection with this press release may contain forward-looking statements, beliefs or opinions regarding Takedas future business, future position and results of operations, including estimates, forecasts, targets and plans for Takeda. In particular, this press release contains forecasts and management estimates related to the financial and operational performance of Takeda, including statements regarding forecasts for Revenue, Operating profit, Adjusted EBITDA, Profit before income taxes, Net profit attributable to owners of Takeda, Basic earnings per share, Amortization and impairment and other income/expense, Underlying Revenue, Underlying Core Earnings margin, Underlying Core EPS and Net Debt. Without limitation, forward looking statements often include the words such as targets, plans, believes, hopes, continues, expects, aims, intends, will, may, should, would, could anticipates, estimates, projects or words or terms of similar substance or the negative thereof. Any forward-looking statements in this document are based on the current assumptions and beliefs of Takeda in light of the information currently available to it. Such forward-looking statements do not represent any guarantee by Takeda or its management of future performance and involve known and unknown risks, uncertainties and other factors, including but not limited to: the economic circumstances surrounding Takedas business, including general economic conditions in Japan, the United States and worldwide; competitive pressures and developments; applicable laws and regulations; the success of or failure of product development programs; decisions of regulatory authorities and the timing thereof; changes in exchange rates; claims or concerns regarding the safety or efficacy of marketed products or products candidates; and post-merger integration with acquired companies, any of which may cause Takedas actual results, performance, achievements or financial position to be materially different from any future results, performance, achievements or financial position expressed or implied by such forward-looking statements. For more information on these and other factors which may affect Takedas results, performance, achievements, or financial position, see Item 3. Key InformationD. Risk Factors in Takedas Registration Statement on Form 20-F filed with the

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Cancer immunology drugs, which harness the bodys immune system to better attack cancer cells, have significantly changed the face of cancer treatment. People with aggressive cancers are now living longer, healthier lives. Unfortunately, cancer immunology therapy only works in a subset of patients.

Now, a new study from scientists at the UCLA Jonsson Comprehensive Cancer Center helps explain why some people with advanced cancer may not respond to one of the leading immunotherapies, PD-1 blockade, and how a new combination approach may help overcome resistance to the immunotherapy drug.

The UCLA study, published today in the inaugural issue of the new scientific journal Nature Cancer, showed that genetic and pharmacological inhibition of the oncogene PAK4 overcomes resistance to anti-PD-1 therapy in preclinical models.

One of the main reasons patients do not respond to PD-1 blockade is because the T cells never make it into the tumor to attack the cancer cells, said lead author Gabriel Abril-Rodriguez, a doctoral candidate in the departments of pharmacology and medicine in the David Geffen School of Medicine at UCLA. We found that biopsies of patients who did not respond to PD-1 blockade showed an overexpression of PAK4, so that led us to believe it played a role in suppressing the immunotherapy treatment.

PAK4 has been known previously to be involved in cell migration and proliferation. The new research from UCLA demonstrates that high expression of this oncogene also correlates with a lack of immune cells migrating into the tumors to destroy the cancer cells.

Using biopsies from people with advanced melanoma who received the immune checkpoint blocking antibody pembrolizumab, UCLA researchers performed RNA sequencing to characterize the phenotype of the tumors. They saw that the tumors that did not respond to PD-1 blockade had a high expression of PAK4 and were not infiltrated by immune cells, meaning that the immune cells had not found their way to the tumor to attack the cancer cells.

The team then inhibited PAK4 in cell lines by either using a drug inhibitor or a gene editing technique called CRISPR-Cas9. The scientists found that deleting PAK4 increased the migration of tumor-specific immune cells and sensitized tumors to PD-1 blockade immunotherapy,reversing the resistance.

Developing new and improved combination treatments like this one for people who do not initially respond to anti-PD-1 treatment is the next step forward in our efforts to make immunotherapy work better for more people, said Dr. Antoni Ribas, the studys senior author, a professor of medicine at the Geffen School and director of the Jonsson cancer centers Tumor Immunology Program. The results from this study could also be expanded to other tumor types that are notoriously resistant to PD-1 blockade, such as pancreatic cancer.

The PAK4 inhibitor used in the study is already being tested in a phase one trial. The combination treatment with anti-PD-1 will be tested in a clinical trial setting in the near future.

The study was funded in part by the National Institutes of Health and the Parker Institute for Cancer Immunotherapy.

Excerpt from:
UCLA study shows inhibition of gene helps overcome resistance to immunotherapy - UCLA Newsroom

Recommendation and review posted by Bethany Smith

To ensure that CRISPR gene editing technology is not misused as was done by a Chinese scientist who created the gene-edited babies recently that attracted global criticism, the Indian Council of Medical Research (ICMR) has framed national guidelines and regulations regarding the procedures and requirements to be followed for performing Gene Therapy in India.

CRISPR stands for clustered regularly interspaced short palindromic repeats are DNA sequences found within the genome of prokaryotes, and are used in genome editing along with enzymes called CRISPR-associated nucleases (most commonly Cas9).

Gene Therapy refers to the process of introduction, removal or change in content of an individuals genetic material with the goal of treating the disease and a possibility of achieving long term cure.

Since this nascent field is emerging in India, the Government has proactively have come up with the National Guidelines for Gene Therapy Product Development and Clinical Trials to promote further research and streamline regulatory processes for future clinical trials using gene therapeutic products (GTPs).

As per the New Drugs and Clinical trial Rules (2019) the GTPs falls under new drug and shall always be deemed to be new drug. Thus as per these rules framed jointly by Indian Council of medical Research and Department of Biotechnology (DBT), academic trials are not applicable to clinical trials using GTPs.

"India has large burden of genetic disorders and unmet medical needs and gene therapy can prove to be a turning point in treatment of such disorders. However, it also brings along with it unique technical risks and ethical challenges," said an official from the ICMR.

She cited the creation of babies using germline gene editing by a Chinese scientist recently that has fuelled a debate on ethical concerns regarding applications of gene therapy technologies.

"This also brought to forefront the requirement of stringent guidelines and regulations to prevent misuse and premature commercialization.

"Many countries around the world have developed rules and guidelines to regulate gene therapy trials.

Taking cognizance of situation, it was felt necessary to frame national guidelines and regulations to direct scientists and clinicians including industry regarding the procedures and requirements to be followed for performing gene therapy in India," she added.

It is proposed to establish Gene Therapy Advisory and Evaluation Committee (GTAEC) anchored at ICMR. GTAEC shall be an independent body of experts representing diverse areas of biomedical research, concerned government agencies and other stakeholders.

"This committee will be composed of a core group of scientists and clinicians in the sector, as well as representation of the government agencies (ICMR, DGHS, CDSCO, DBT, DST, MCI). For each disease area in GTP trials, specific clinical consultants with extensive disease specific expertise will be co-opted to aid in the decision-making process," as per the guidelines.

It says that biological material from humans can be procured only from clinics/hospitals that have an evaluation committee.

The EC must ensure that the Standard Operating Procedures (SOPs) are in compliance with the national guidelines. "Investigators should treat the biological material with utmost respect and adequate care to avoid its misuse. The institute needs to define SOPs for development, production; storage and disposal of the GTPs or its components should be as per the Regulations and Guidelines on Bio-safety of Recombinant DNA Research and Bio-containment 2017," says the guidelines.

Until 2017, almost 2600 gene therapy and 6 clinical trials have been conducted worldwide in 38 countries, of which 64.9 per cent were in USA, 23.2 per cent in Europe and approximately 6.5 per cent were in Asia.

Within Asia, China has reported about 84 gene therapy clinical trials, followed by Japan (44 trials) and South Korea (14 trials). The vast majority of gene therapy trials have addressed cancer (66.6 per cent), monogenic diseases (11.5 per cent), cardiovascular diseases (6.2 per cent) and infectious diseases (6.3 per cent)

Link:
ICMR issues guidelines for gene therapy in India - Daily Pioneer

Recommendation and review posted by Bethany Smith

BOSTON and LONDON, Dec. 08, 2019 (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a leading commercial-stage biopharmaceutical company dedicated to transforming the lives of patients with serious and life-threatening rare diseases through innovative gene therapies, will be presenting new registrational data from multiple programs at the 61st American Society of Hematology (ASH) Annual Meeting being held December 7-10, 2019 in Orlando, FL.

On Sunday, December 8, 2019, investigators will describe ongoing clinical progress for two lead development programs in the companys primary immune deficiencies portfolio: OTL-103, an investigational gene therapy in development for the treatment of Wiskott-Aldrich syndrome (WAS) at the San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget) in Milan, Italy; and OTL-101, an investigational gene therapy in development for the treatment of adenosine deaminase severe combined immunodeficiency (ADA-SCID).

In addition, on Monday, December 9, 2019, investigators will deliver an oral presentation featuring updated data from the ongoing clinical proof-of-concept study of OTL-203, an investigational gene therapy in development for the treatment of mucopolysaccharidosis type I (MPS-I) at SR-Tiget.

To learn more about Orchards approach to ex vivo, autologous, hematopoietic stem cell (HSC) based gene therapy, conference attendees can visit booth #2228 in the Exhibition Hall.

Full presentation details are below:

Poster Presentation Details

Lentiviral Hematopoietic Stem and Progenitor Cell Gene Therapy for Wiskott-Aldrich Syndrome (WAS): Up to 8 Years of Follow up in 17 Subjects Treated Since 2010Publication Number: 3346Session: 801. Gene Therapy and Transfer: Poster IIDate and time: Sunday, December 8, 6:00-8:00pm ET

Lentiviral Gene Therapy with Autologous Hematopoietic Stem and Progenitor Cells (HSPCs) for the Treatment of Severe Combined Immune Deficiency Due to Adenosine Deaminase Deficiency (ADA-SCID): Results in an Expanded CohortPublication Number: 3345Session: 801. Gene Therapy and Transfer: Poster IIDate and time: Sunday, December 8, 6:00-8:00pm ET

Oral Presentation Details

Extensive Metabolic Correction of Hurler Disease by Hematopoietic Stem Cell-Based Gene Therapy: Preliminary Results from a Phase I/II TrialPublication Number: 607Session: 801. Gene Therapy and Transfer: Gene Therapies for Non-Malignant DisordersDate and time: Monday, December 9, 7:00am ET

About ADA-SCID and OTL-101Severe combined immune deficiency due to adenosine deaminase deficiency (ADA-SCID) is a rare, life-threatening, inherited disease of the immune system caused by mutations in the ADA gene resulting in a lack of, or minimal, immune system development.1-4 The first symptoms of ADA-SCID typically manifest during infancy with recurrent severe bacterial, viral and fungal infections and overall failure to thrive, and without treatment the condition can be fatal within the first two years of life. The incidence of ADA-SCID is currently estimated to be one in 500,000 live births in the United States and between one in 200,000 and one in 1 million in Europe.3 OTL-101 is an autologous, ex vivo, hematopoietic stem cell-based gene therapy for the treatment of patients diagnosed with ADA-SCID being investigated in multiple clinical trials in the United States and Europe, including a registrational trial at the University of California, Los Angeles (UCLA). OTL-101 has received orphan drug designation from the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) for the treatment of ADA-SCID, and Breakthrough Therapy Designation from the FDA.

About WAS and OTL-103Wiskott-Aldrich Syndrome (WAS) is a life-threatening inherited immune disorder characterized by autoimmunity and abnormal platelet function and manifests with recurrent, severe infections and severe bleeding episodes, which are the leading causes of death in this disease. Without treatment, the median survival for WAS patients is 14 years of age. Treatment with stem cell transplant carries significant risk of mortality and morbidities. OTL-103 is an ex vivo, autologous, hematopoietic stem cell-based gene therapy developed for the treatment of WAS that Orchard acquired from GSK in April 2018 and has been developed at the San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget) in Milan, Italy. The global incidence of WAS is estimated to be about 100-260 births per year, with a global prevalence of 2,900-4,700 patients.

About MPS-I and OTL-203Mucopolysaccharidosis type I (MPS-I) is a rare inherited neurometabolic disease caused by a deficiency of the IDUA (alpha-L-iduronidase) lysosomal enzyme required to break down glycosaminoglycans (also known as GAGs or mucopolysaccharides). The accumulation of GAGs across multiple organ systems results in the symptoms of MPS-I including neurocognitive impairment, skeletal deformity, loss of vision and hearing, hydrocephalus, and cardiovascular and pulmonary complications. MPS-I occurs at an overall estimated frequency of one in every 100,000 live births.5 There are three subtypes of MPS-I; approximately 60 percent of MPS-I patients have the severe Hurler subtype and, when untreated, these patients rarely live past the age of 10.Id Treatment options for MPS-I include hematopoietic stem cell transplant and chronic enzyme replacement therapy, both of which have significant limitations. Though early intervention with enzyme replacement therapy has been shown to delay or prevent some clinical features of the condition, it has only limited efficacy on neurological symptoms. OTL-203 is an ex vivo, autologous, hematopoietic stem cell-based gene therapy being studied for the treatment of MPS-I. Orchard was granted an exclusive worldwide license to intellectual property rights to research, develop, manufacture and commercialize the gene therapy program for the treatment of MPS-I developed by the San Raffaele-Telethon Institute for Gene Therapy in Milan, Italy.

About Orchard Orchard Therapeutics is a fully integrated commercial-stage biopharmaceutical company dedicated to transforming the lives of patients with serious and life-threatening rare diseases through innovative gene therapies.

Orchards portfolio of ex vivo, autologous, hematopoietic stem cell (HSC) based gene therapies includes Strimvelis, a gammaretroviral vector-based gene therapy and the first such treatment approved by the European Medicines Agency for severe combined immune deficiency due to adenosine deaminase deficiency (ADA-SCID). Additional programs for neurometabolic disorders, primary immune deficiencies and hemoglobinopathies are all based on lentiviral vector-based gene modification of autologous HSCs and include three advanced registrational studies for metachromatic leukodystrophy (MLD), ADA-SCID and Wiskott-Aldrich syndrome (WAS), clinical programs for X-linked chronic granulomatous disease (X-CGD), transfusion-dependent beta-thalassemia (TDT) and mucopolysaccharidosis type I (MPS-I), as well as an extensive preclinical pipeline. Strimvelis, as well as the programs in MLD, WAS and TDT were acquired by Orchard from GSK in April 2018 and originated from a pioneering collaboration between GSK and the San Raffaele Telethon Institute for Gene Therapy in Milan, Italy initiated in 2010.

Orchard currently has offices in the UK and the U.S., including London, San Francisco and Boston.

Forward-Looking StatementsThis press release contains certain forward-looking statements about Orchards strategy, future plans and prospects, which are made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements may be identified by words such as anticipates, believes, expects, intends, projects, and future or similar expressions that are intended to identify forward-looking statements. Forward-looking statements include express or implied statements relating to, among other things, the therapeutic potential of Orchards product candidates, including the product candidate or candidates referred to in this release, Orchards expectations regarding the timing of regulatory submissions for approval of its product candidates, including the product candidate or candidates referred to in this release, the timing of announcement of clinical data for its product candidates and the likelihood that such data will be positive and support further clinical development and regulatory approval of these product candidates, including any cryopreserved formulations of such product candidates, and the likelihood of approval of such product candidates by the applicable regulatory authorities. These statements are neither promises nor guarantees and are subject to a variety of risks and uncertainties, many of which are beyond Orchards control, which could cause actual results to differ materially from those contemplated in these forward-looking statements. In particular, the risks and uncertainties include, without limitation: the risk that any one or more of Orchards product candidates, including the product candidate or candidates referred to in this release, will not be successfully developed or commercialized, the risk of cessation or delay of any of Orchards ongoing or planned clinical trials, the risk that prior results, such as signals of safety, activity or durability of effect, observed from preclinical studies or clinical trials will not be replicated or will not continue in ongoing or future studies or trials involving Orchards product candidates, the delay of any of Orchards regulatory submissions, the failure to obtain marketing approval from the applicable regulatory authorities for any of Orchards product candidates, the receipt of restricted marketing approvals, and the risk of delays in Orchards ability to commercialize its product candidates, if approved. Given these uncertainties, the reader is advised not to place any undue reliance on such forward-looking statements.

Other risks and uncertainties faced by Orchard include those identified under the heading "Risk Factors" in Orchards annual report on Form 20-F for the year ended December 31, 2018 as filed with the U.S. Securities and Exchange Commission (SEC) on March 22, 2019, as well as subsequent filings and reports filed with the SEC. The forward-looking statements contained in this press release reflect Orchards views as of the date hereof, and Orchard does not assume and specifically disclaims any obligation to publicly update or revise any forward-looking statements, whether as a result of new information, future events or otherwise, except as may be required by law.

1Orphanet. SCID due to ADA deficiency. 2Whitmore KV, Gaspar HB. Front Immunol. 2016;7:314. 3Kwan A, et al. JAMA. 2014;312:729-738. 4Sauer AV, et al. Front Immunol. 2012;3:265. 5Beck et al. The Natural History of MPS I: Global Perspectives from the MPS I Registry. Genetics in Medicine 2014, 16(10), 759.

Contacts

InvestorsRenee LeckDirector, Investor Relations+1 862-242-0764Renee.Leck@orchard-tx.com

MediaMolly CameronManager, Corporate Communications+1 978-339-3378media@orchard-tx.com

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Orchard Therapeutics Showcases Clinical Data at the 61st American Society of Hematology Annual Meeting - BioSpace

Recommendation and review posted by Bethany Smith