Drinking certain energy drinks may cause significant damage to the heart, according to new findings published in Food and Chemical Toxicology.

Because the consumption of these beverages is not regulated and they are widely accessible over the counter to all age groups, the potential for adverse health effects of these products is a subject of concern and needed research, lead researcher Ivan Rusyn, MD, PhD, a professor at Texas A&M University in College Station, said in a prepared statement.

Rusyn et al. assessed a total of 17 popular energy drinks, studying their chemical profiles and looking for any associations with potential cardiac complications. Energy drinks sold by Adrenaline, Shoc, Bang Star, C4, CELSIUS, HEAT, EBOOST, Game Fuel, GURU, Kill Cliff, Kickstart, Monster Energy, Red Bull, Reign, Rockstar, RUNA, UPTIME, Venom Energy and Xyience Energy were all part of the teams analysis.

Overall, the authors found that stem cell-derived cardiomyocyteshuman heart cells grown in a laboratoryshowed signs of an increased beat rate after being exposed to some energy drinks. Also, theophylline, adenine and azelate were all ingredients the team associated with potentially contributing to QT prolongation in cardiomyocytes.

Go here to see the original:
Energy drinks may damage the heart, researchers warnshould the FDA get involved? - Cardiovascular Business

Recommendation and review posted by Bethany Smith

Feb. 16, 2021 12:00 UTC

CAMBRIDGE, Mass.--(BUSINESS WIRE)-- bluebird bio, Inc. (Nasdaq: BLUE) announced today that the company has placed its Phase 1/2 (HGB-206) and Phase 3 (HGB-210) studies of LentiGlobin gene therapy for sickle cell disease (SCD) (bb1111) on a temporary suspension due to a reported Suspected Unexpected Serious Adverse Reaction (SUSAR) of acute myeloid leukemia (AML).

In line with the clinical study protocols for HGB-206 and HGB-210, bluebird bio placed the studies on temporary suspension following a report received last week that a patient who was treated more than five years ago in Group A of HGB-206 was diagnosed with AML. The company is investigating the cause of this patients AML in order to determine if there is any relationship to the use of BB305 lentiviral vector in the manufacture of LentiGlobin gene therapy for SCD. In addition, a second SUSAR of myelodysplastic syndrome (MDS) in a patient from Group C of HGB-206 was reported last week to the company and is currently being investigated.

No cases of hematologic malignancy have been reported in any patient who has received treatment with betibeglogene autotemcel for transfusion-dependent -thalassemia (licensed as ZYNTEGLOTM in the European Union and the United Kingdom), however because it is also manufactured using the same BB305 lentiviral vector used in LentiGlobin gene therapy for SCD, the company has decided to temporarily suspend marketing of ZYNTEGLO while the AML case is assessed.

The safety of every patient who has participated in our studies or is treated with our gene therapies is the utmost priority for us, said Nick Leschly, chief bluebird. We are committed to fully assessing these cases in partnership with the healthcare providers supporting our clinical studies and appropriate regulatory agencies. Our thoughts are with these patients and their families during this time.

The independent safety review board monitoring the companys studies as well as the U.S. Food and Drug Administration (FDA) and European Medicines Agency (EMA) have been advised of these cases and bluebird bio will continue to work with regulatory agencies to complete its investigation.

Investor Conference Call Information

bluebird bio will hold a conference call to discuss this update on Tuesday, February 16 at 8:00 a.m. ET. Investors may listen to the call by dialing (844) 825-4408 from locations in the United States or +1 (315) 625-3227 from outside the United States. Please refer to conference ID number 880-6406.

To access the live webcast of bluebird bios presentation, please visit the Events & Presentations page within the Investors & Media section of the bluebird bio website at http://investor.bluebirdbio.com. A replay of the webcast will be available on the bluebird bio website for 90 days following the event.

About HGB-206 and HGB-210

HGB-206 is an ongoing, Phase 1/2 open-label study designed to evaluate the efficacy and safety of LentiGlobin gene therapy for sickle cell disease (SCD) that includes three treatment cohorts: Groups A, B and C. A refined manufacturing process designed to increase vector copy number (VCN) and further protocol refinements made to improve engraftment potential of gene-modified stem cells were used for Group C. Group C patients also received LentiGlobin for SCD made from HSCs collected from peripheral blood after mobilization with plerixafor, rather than via bone marrow harvest, which was used in Groups A and B of HGB-206.

HGB-210 is an ongoing Phase 3 single-arm open-label study designed to evaluate the efficacy and safety of LentiGlobin gene therapy for SCD in patients between two years and 50 years of age with sickle cell disease.

About LentiGlobin for SCD (bb1111)

LentiGlobin gene therapy for sickle cell disease (bb1111) is an investigational treatment being studied as a potential treatment for SCD. bluebird bios clinical development program for LentiGlobin for SCD includes the completed Phase 1/2 HGB-205 study, the ongoing Phase 1/2 HGB-206 study, and the ongoing Phase 3 HGB-210 study.

The U.S. Food and Drug Administration granted orphan drug designation, fast track designation, regenerative medicine advanced therapy (RMAT) designation and rare pediatric disease designation for LentiGlobin for SCD.

LentiGlobin for SCD received orphan medicinal product designation from the European Commission for the treatment of SCD, and Priority Medicines (PRIME) eligibility by the European Medicines Agency (EMA) in September 2020.

bluebird bio is conducting a long-term safety and efficacy follow-up study (LTF-307) for people who have participated in bluebird bio-sponsored clinical studies of LentiGlobin for SCD. For more information visit: https://www.bluebirdbio.com/our-science/clinical-trials or clinicaltrials.gov and use identifier NCT04628585 for LTF-307.

LentiGlobin for SCD is investigational and has not been approved in any geography.

About ZYNTEGLO (betibeglogene autotemcel)

Betibeglogene autotemcel (beti-cel) is a one-time gene therapy that adds functional copies of a modified form of the -globin gene (A-T87Q-globin gene) into a patients own hematopoietic (blood) stem cells (HSCs). Once a patient has the A-T87Q-globin gene, they have the potential to produce HbAT87Q, which is gene therapy-derived adult Hb, at levels that may eliminate or significantly reduce the need for transfusions. In studies of beti-cel, transfusion independence (TI) is defined as no longer needing red blood cell transfusions for at least 12 months while maintaining a weighted average Hb of at least 9 g/dL.

The European Commission granted conditional marketing authorization (CMA) for beti-cel, marketed as ZYNTEGLO gene therapy, for patients 12 years and older with transfusion-dependent -thalassemia (TDT) who do not have a 0/0 genotype, for whom hematopoietic stem cell (HSC) transplantation is appropriate, but a human leukocyte antigen (HLA)-matched related HSC donor is not available.

Non-serious adverse events (AEs) observed during clinical studies that were attributed to beti-cel included abdominal pain, thrombocytopenia, leukopenia, neutropenia, hot flush, dyspnea, pain in extremity, tachycardia and non-cardiac chest pain. One serious adverse event (SAE) of thrombocytopenia was considered possibly related to beti-cel.

Additional AEs observed in clinical studies were consistent with the known side effects of HSC collection and bone marrow ablation with busulfan, including SAEs of veno-occlusive disease.

For details, please see the Summary of Product Characteristics (SmPC).

On April 28, 2020, the European Medicines Agency (EMA) renewed the CMA for beti-cel. The CMA for beti-cel is valid in the 27 member states of the EU as well as the UK, Iceland, Liechtenstein and Norway.

The U.S. Food and Drug Administration granted beti-cel Orphan Drug status and Breakthrough Therapy designation for the treatment of TDT. Beti-cel is not approved in the U.S. Beti-cel continues to be evaluated in the ongoing Phase 3 Northstar-2 (HGB-207) and Northstar-3 (HGB-212) studies.

bluebird bio is conducting a long-term safety and efficacy follow-up study, LTF-303 for people who have participated in bluebird bio-sponsored clinical studies of ZYNTEGLO.

About bluebird bio, Inc.

bluebird bio is pioneering gene therapy with purpose. From our Cambridge, Mass., headquarters, were developing gene and cell therapies for severe genetic diseases and cancer, with the goal that people facing potentially fatal conditions with limited treatment options can live their lives fully. Beyond our labs, were working to positively disrupt the healthcare system to create access, transparency and education so that gene therapy can become available to all those who can benefit.

bluebird bio is a human company powered by human stories. Were putting our care and expertise to work across a spectrum of disorders: cerebral adrenoleukodystrophy, sickle cell disease, -thalassemia and multiple myeloma, using gene and cell therapy technologies including gene addition, and (megaTAL-enabled) gene editing.

bluebird bio has additional nests in Seattle, Wash.; Durham, N.C.; and Zug, Switzerland. For more information, visit bluebirdbio.com.

Follow bluebird bio on social media: @bluebirdbio, LinkedIn, Instagram and YouTube.

ZYNTEGLO, betibeglogene autotemcel, beti-cel, and bluebird bio are trademarks of bluebird bio, Inc.

Forward-Looking Statements

This release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, including statements regarding the Companys timing and expectations regarding its investigation of the relationship of the AML and MDS events to the use of lentiviral vector BB305 in LentiGlobin gene therapy for SCD, and any myeloablation regimen used in connection with treatment. Any forward-looking statements are based on managements current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements, many of which are beyond the Companys control. These risks and uncertainties include, but are not limited to: the risk that the Company may not be able to definitively determine whether the lentiviral vector BB305 used in LentiGlobin gene therapy for SCD and in betibeglogene autotemcel is related to the patients AML in a timely manner, or at all; the risk that the lentiviral vector BB305 has caused insertional oncogenic events, including AML; the risk that insertional oncogenic events associated with lentiviral vector or additional MDS events associated with myeloablation will be discovered or reported over time; the risk that regulatory authorities may impose a clinical hold, in addition to our temporary clinical hold on the HGB-206 and HGB-210 studies, or on additional programs; the risk that we may not be able to address regulatory authorities concerns quickly or at all; the risk that we may not resume patient treatment with ZYNTEGLO in the commercial context in a timely manner or at all; the risk that our lentiviral vector platform across our severe genetic disease programs may be implicated, affecting the development and potential approval of elivaldogene autotemcel; the risk that we may not be able to execute on our business plans, including our commercialization plans, meeting our expected or planned regulatory milestones, submissions, and timelines, research and clinical development plans, and in bringing our product candidates to market; and the risk that with the impact on the execution and timing of our business plans, we may not successfully execute our previously announced plans to spin off our oncology programs into an independent publicly-traded entity. For a discussion of other risks and uncertainties, and other important factors, any of which could cause our actual results to differ from those contained in the forward-looking statements, see the section entitled Risk Factors in our most recent Form 10-Q, as well as discussions of potential risks, uncertainties, and other important factors in our subsequent filings with the Securities and Exchange Commission. All information in this press release is as of the date of the release, and bluebird bio undertakes no duty to update this information unless required by law.

View source version on businesswire.com: https://www.businesswire.com/news/home/20210216005442/en/

Read the original:
bluebird bio Announces Temporary Suspension on Phase 1/2 and Phase 3 Studies of LentiGlobin Gene Therapy for Sickle Cell Disease (bb1111) - BioSpace

Recommendation and review posted by Bethany Smith

Betibeglogene autotemcel (beti-cel), a one-time gene therapy, enabled durable transfusion independence in most patients with transfusion-dependent -thalassemia (TDT) who were treated across 4 clinical studies.

Of 60 patients enrolled overall, 17 of 22 (77%) treated in the 2 phase 1/2 studies were able to stop packed red blood cell transfusions. In the 2 phase 3 studies, which used a refined manufacturing process resulting in improved beti-cel characteristics, 89% (n = 31/35) of patients with at least 6 months of follow-up achieved transfusion independence for more than 6 months,1 reported Suradej Hongeng, MD, during the virtual 2021 Transplantation & Cellular Therapy Meetings.

The median follow-up after beti-cel infusion in the 4 studies has been 24.8 months (range, 1.1-71.8).

With up to 6 years of follow-up, 1-time beti-cel gene therapy enabled durable transfusion independence in the majority of patients, said Hongeng, from Ramathibodi Hospital of Mahidol University, in Bangkok, Thailand.

Patients who achieved transfusion independence experienced a 38% median reduction in liver iron concentration (LIC) from baseline to month 48. The median reduction in LIC was 59% in patients with a baseline LIC more than 15 mg/g dw. A total of 21 of 37 (57%) patients who achieved transfusion independence have stopped iron chelation for 6 months or longer, with a median duration of 18.5 months from stopping iron chelation to last follow-up.

Erythropoiesis as determined by soluble transferrin receptor level was also improved in transfusion-independent patients. Bone marrow biopsies showed improvement in the myeloid:erythroid ratio.

Beti-cel adds functional copies of a modified form of the -globin (A-T87Q-globin) gene into a patients own hematopoietic stem cells (HSCs) through transduction of autologous CD34+ cells using a BB305 lentiviral vector. Following single-agent busulfan myeloablative conditioning, beti-cel is infused, after which the transduced HSCs engraft and reconstitute red blood cells containing functional adult hemoglobin derived from the gene therapy.

Of the 60 patients treated, 43 were genotype non-/ and 17 were / . The median age at consent was 20 years in the phase 1/2 trials and 15 years in the phase 3 trials. Median LIC at baseline was 7.1 and 5.5 mg Fe/g dw, respectively, and median cardiac T2 was 34 and 37 msec, respectively. The vector copy number was 0.8 in the phase 1/2 trial and 3.0 in the phase 3 study. Additionally, 32t and 78t CD34+ cells were transduced, respectively.

The phase 1/2 studies showed promising results but lower achievement of transfusion independence in patients with the / genotype, leading to a refinement in the manufacturing process, which resulted in a higher number of transduced cells and a higher number of vector copy number, said Hongeng.

The median time to neutrophil engraftment was 22.5 days and the median time to platelet engraftment was 44 days. Lymphocyte subsets were generally within the normal range after beti-cel infusion, which is different from allogeneic stem cell [transplantation], which is probably around 6 months to a year to get complete recovery of immune reconstitution, he said. The median duration of hospitalization was 42 days.

All patients were alive at the last follow-up (March 3, 2020). Eleven of 60 (18%) of patients experienced at least 1 adverse event (AE) considered related or possibly related to beti-cel, the most common being abdominal pain (8%) and thrombocytopenia (5%). Serious AEs were those expected after myeloablative conditioning: veno-occlusive liver disease (8%), neutropenia (5%), pyrexia (5%), thrombocytopenia (5%), and appendicitis, febrile neutropenia, major depression, and stomatitis (3% each).

Of the 7 patients experiencing veno-occlusive liver disease, 3 were of grade 4 and 2 were of grade 3. Two other patients had grade 2 veno-occlusive disease. There were no cases of insertional oncogenesis.

Persistent vector-positive hematopoietic cells and durable HbaT87Q levels supported stable total hemoglobin over time. In phase 3 trials, the median peripheral blood vector copy number was 1.2 c/dg at month 12 and 2.0 c/dg at month 24, and the median total hemoglobin was 11.5 g/dL at month 12 and 12.9 g/dL at month 24.

The weighted average of hemoglobin during transfusion independence in the phase 1/2 trials was 10.4 g/dL, and patients were transfusion-independent for a median of 51.2 months. In the phase 3 studies, the weighted average of hemoglobin during transfusion independence was 11.9 g/dL, and patients were transfusion-independent for a medium 17.7 months.

Hongeng S, Thompson AA, Kwiatkowski JL, et al. Efficacy and safety of betibeglogene autotemcel (beti-cel; LentiGlobin for -thalassemia) gene therapy in 60 patients with transfusion-dependent -thalassemia (TDT) followed for up to 6 years post-infusion. Presented at: 2021 Transplantation & Cellular Therapy Meetings; February 8-12, 2021; virtual. Abstract 1.

Read this article:
Beti-Cel Gene Therapy Frees Patients With Beta-Thalassemia From Red Blood Cell Transfusions - OncLive

Recommendation and review posted by Bethany Smith

CRISPR Therapeutics (CRSP) is one of my favorite biotech companies as the big leader among gene-editing pioneers.

But I had to let the stock go -- right before shares launched into the December ASH meeting (American Society of Hematology) -- because analysts were so bearish on the outlook for when the R&D pipeline would produce any revenues, much less profits.

Of course, talking about revenues and profits for world-changing, early-stage medical science is almost always a non sequitur.

Still in late summer, I let go of my CRSP shares for a 71% gain. But as I describe in this September video and article, it was not the first, nor the last, of great trading gains in the greatest of CRISPR companies...

CRISPR Stocks: Buy or Trade?

In fact, here's the actual trading record from my Healthcare Innovators portfolio of my previous 3 CRSP trades...

So why did CRSP launch from $110 to $210 in December and January?

It was mostly about investors recognizing that the company's early data in treating debilitating illnesses like Sickle Cell Disease (SCD) could indeed become world-changing for millions afflicted with the genetic impairment to their red blood cells.

SCD comprises a group of disorders that cause red blood cells to become misshapen and break down. Red blood cells contort into a sickle shape, and die early, leaving a shortage of healthy red blood cells (sickle cell anemia), and can block blood flow causing pain (sickle cell crisis). Infections, pain, and fatigue are symptoms of sickle cell disease. Current treatments include frequent medications, blood transfusions and, in extreme cases, a bone-marrow transplant -- but no cures.

As Antonio Regalado wrote in the MIT Technology Review wrote last week, "The burden of sickle-cell, an inherited disease that shortens lives by decades (or, in poor regions, kills during childhood), falls most heavily on Black people in equatorial Africa, Brazil, and the US. HIV has also become a lingering scourge: about two-thirds of people living with the virus, or dying from it, are in Africa."

I explained some of this potential in this vlog on December 10...

CRISPR Gene Editing: Owning the Future of Medicine

A secondary "igniter" of all CRISPR stocks launching higher in Dec-Jan (besides my video commentaries linked above) was the investment activity of Cathie Wood and her revolutionary ETF firm ARK Invest.

I produced a video and article about her one-woman investor revolution in early January with her monster ETFs ARK Innovation (ARKK) and ARK Genomics (ARKG)...

How Cathie Put the Wood to Wall Street: TSLA, SQ, ROKU, CRSP, BIDU

There will be a time to buy CRSP again. But as with all emerging Biotechs, you sometimes have to wait for the next clinical data catalyst -- or an M&A one.

I'm betting the latter is the sooner driver of the next move from a $10 billion market cap to a $20 billion one.

Meanwhile, I own the smaller pair of Editas Medicine (EDIT) and Intellia Therapeutics (NTLA) near $5 billion.

Stay CRSPy!

Cooker

Kevin Cook is a Senior Stock Strategist for Zacks Investment Research where he runs the Healthcare Innovators portfolio.

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Intellia Therapeutics, Inc. (NTLA) : Free Stock Analysis Report

CRISPR Therapeutics AG (CRSP) : Free Stock Analysis Report

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Zacks Investment Research

The rest is here:
Bear of the Day: CRISPR Therapeutics (CRSP) - Yahoo Finance

Recommendation and review posted by Bethany Smith

For Immediate Release

Chicago, IL February 11, 2021 Zacks Equity Research Shares of Meridian Bioscience, Inc. VIVO as the Bull of the Day, CRISPR Therapeutics AG CRSP as the Bear of the Day. In addition, Zacks Equity Research provides analysis on Tilray, Inc. TLRY and Uber Technologies, Inc. UBER.

Here is a synopsis of all four stocks:

Meridian Bioscienceis a $1 billion provider of diagnostic test kits for gastrointestinal and respiratory infectious diseases. The company is expected to grow sales 32% this year to $335 million.

And after reporting a strong beat-and-raise quarter last week, analysts had to boost their EPS estimates over 30% from $1.24 to $1.63. So VIVO is back to a Zacks #1 Rank, sporting a projected 52% rise in profits.

I wrote about VIVO in early January and said it wasn't too late for investors to grab hold of this profit rocket near $20 per share...

"Bottom line on VIVO: I always pay attention to small companies growing their sales rapidly as they could become acquisition targets by larger biopharma or MedTech players. Buying VIVO near $20 offers excellent risk/reward, with or without an M&A suitor."

And then last week, right before the company report, I produced a video and article where I talked about why the COVID-19 testing stocks were under-appreciated given their fantastic growth...

Biotech Bonanza: COVID Launches Science at Warp Speed

Well here we are as VIVO pushed to 13-year highs above $30 with the earnings surprise and strong upside guidance.

Following the quarterly report, Piper Sandler analyst Steven Mah, a consistently bullish VIVO fan, raised his price target on Meridian Bioscience to $34 from $26 and reiterated an Overweight rating. Mah believes the company guidance was once again "very conservative" citing management's "pragmatic approach given the limited visibility on the durability of COVID-19 tailwinds."

Mah explained his increased confidence in Meridian's longer-term COVID-19 tailwinds given the Biden Administration's testing stance, slower than expected vaccine rollout, and new strain emergence.

Story continues

Meridian Bioscience develops, manufactures, distributes, and sells diagnostic test kits primarily for gastrointestinal and respiratory infectious diseases, and elevated blood lead levels worldwide. The company operates through Diagnostics and Life Science segments. They describe their mission as helping providers make better diagnostic decisions with a focus on gastrointestinal, neonatal, pediatrics, and respiratory conditions.

The Diagnostics segment offers testing platforms, including real-time PCR (polymerase chain reaction) amplification under the Revogene brand; isothermal DNA amplification under the Alethia brand; lateral flow immunoassay using fluorescent chemistry under the Curian brand; rapid immunoassay under the ImmunoCard and ImmunoCard STAT! brands; enzyme-linked immunoassays under the PREMIER brand; anodic stripping voltammetry under the LeadCare and PediaStat brands; and urea breath testing for H. pylori under the BreathID brand.

I have written often in the past few months of specialized diagnostic companies likeQuidelandHologicas they build new revenue streams from SARS-CoV-2 testing. These revenue streams are likely sustainable as the virus mutates and requires modified tests.

And I recently bought shares of VIVO for the Zacks Healthcare Innovators portfolio because I liked the growth outlook for this small player in a rapidly expanding market for rapid diagnostics -- including coronavirus testing which will continue to be part of our lives for years to come, even with vaccines.

While Meridian Bioscience is a David among diagnostic Goliaths, its long and fascinating history surprised me. From the company website...

In 1977, Bill Motto founded Meridian Bioscience on a $500 investment in his Cincinnati homes basement. Meridians first product was distributing a rapid fungal test developed by the University of Kentucky. While calling on his hospital and research customers, Bill noticed there was no easy, clean way to transport patient samples. He developed the innovative Para-Pak stool transport system to meet this need.

As the product line grew, so did Meridians research and development, leading to a breakthrough in 1982 with a 10-minute rapid test for strep throat. Before the Meridian test, doctors would have to wait for two to three days for a culture result. Innovation continued as the company brought several cutting edge diagnostic technologies to market, including a DNA testing platform and first-of-their-kind tests for C. difficile, E. coli, H. pylori amongst others.

The new bottom line on VIVO:I continue to hold the shares and would recommend new positions between $25 and $27 looking for new bull market highs above $30 by June.

Disclosure: I own shares of QDEL, HOLX, and VIVO for the Zacks Healthcare Innovators portfolio.

CRISPR Therapeuticsis one of my favorite biotech companies as the big leader among gene-editing pioneers.

But I had to let the stock go -- right before shares launched into the December ASH meeting (American Society of Hematology) -- because analysts were so bearish on the outlook for when the R&D pipeline would produce any revenues, much less profits.

Of course, talking about revenues and profits for world-changing, early-stage medical science is almost always a non sequitur.

Still in late summer, I let go of my CRSP shares for a 71% gain. But as I describe in this September video and article, it was not the first, nor the last, of great trading gains in the greatest of CRISPR companies...

CRISPR Stocks: Buy or Trade?

So why did CRSP launch from $110 to $210 in December and January?

It was mostly about investors recognizing that the company's early data in treating debilitating illnesses like Sickle Cell Disease (SCD) could indeed become world-changing for millions afflicted with the genetic impairment to their red blood cells.

SCD comprises a group of disorders that cause red blood cells to become misshapen and break down. Red blood cells contort into a sickle shape, and die early, leaving a shortage of healthy red blood cells (sickle cell anemia), and can block blood flow causing pain (sickle cell crisis). Infections, pain, and fatigue are symptoms of sickle cell disease. Current treatments include frequent medications, blood transfusions and, in extreme cases, a bone-marrow transplant -- but no cures.

As Antonio Regalado wrote in the MIT Technology Review wrote last week, "The burden of sickle-cell, an inherited disease that shortens lives by decades (or, in poor regions, kills during childhood), falls most heavily on Black people in equatorial Africa, Brazil, and the US. HIV has also become a lingering scourge: about two-thirds of people living with the virus, or dying from it, are in Africa."

I explained some of this potential in this vlog on December 10...

CRISPR Gene Editing: Owning the Future of Medicine

A secondary "igniter" of all CRISPR stocks launching higher in Dec-Jan (besides my video commentaries linked above) was the investment activity of Cathie Wood and her revolutionary ETF firm ARK Invest.

I produced a video and article about her one-woman investor revolution in early January with her monster ETFsARK Innovation:

How Cathie Put the Wood to Wall Street: TSLA, SQ, ROKU, CRSP, BIDU

There will be a time to buy CRSP again. But as with all emerging Biotechs, you sometimes have to wait for the next clinical data catalyst -- or an M&A one.

I'm betting the latter is the sooner driver of the next move from a $10 billion market cap to a $20 billion one.

Stay CRSPy!

Cooker

Kevin Cook is a Senior Stock Strategist for Zacks Investment Research where he runs the Healthcare Innovators portfolio.

Markets continued to hover close to the zero-line as of Wednesdays close, with just the Dow finishing in the green among major indexes. The blue-chips rose 0.20% for a new all-time high, while the Nasdaq, S&P 500 and Russell 2000 all took a breather: -0.25%, -0.03% and -0.72% on the day.

One of the major pot stocks we discussed yesterday in this space,Tilray, continues its big run in what looks more like the latest short-squeeze stock, with Reddit groups now piling into the marijuana-based pharmaceutical company of late. Shares shot up another 51% Wednesday, following a +44% performance yesterday; Tilray is now up 400% in just the last month alone.

For sure, increased acceptance in U.S. states and countries around the world are a reason for the stock to do well. However, its market cap has more than doubled and the company has no P/E because it is forecast for negative earnings both in the upcoming quarterly report and full fiscal year. Tilray is up another 10% in late trading, up near $71 per share. This stock was trading at $19 per share on February 1st.

Uber followed a nice 6% bump in regular-day Wednesday trading with a worse-than-expected fiscal Q3 report, missing on the bottom line by a penny to -54 cents per share (though still better than the -64 cents the company reported in the year-ago quarter). Revenues grew $3.17 billion in the quarter, well off the expected pace of $3.55 billion in the Zacks consensus. Yet shares are only selling off minimally in the after-market; the company still says its on track to its profitability goal in 2021.

Though Uber posted a net loss per year of $6.8 billion on ride-sharing revenues down 52% year over year, its Uber Eats delivery service grew 224% in its fiscal Q3. Monthly active platform consumers gained a million more than predicted in the quarter, +93 million. And when one figures in the ride-sharing comeback seemingly inevitable as the Covid-19 pandemic is finally beaten back with vaccinations, we see that Uber looks to have weathered its worst-possible storm and survived.For more on UBER's earnings, click here.

Speaking of the coronavirus, nearly 45 million vaccination doses have now been administered, and the post-holiday season peak now looks to have been successfully scaled. We are now back to 7-day case rates back where they were in October and, importantly, pointed in the right direction. More than 27 million Americans have reportedly contracted Covid-19, leading to more than 466K fatalities. More good news: the death rate is now flat for those whove gotten the worst of the disease.

Questions or comments about this article and/or its author? Click here>>

Experts extracted 7 stocks from the list of 220 Zacks Rank #1 Strong Buys that have beaten the market more than 2X over with a stunning average gain of +24.9% per year.

These 7 were selected because of their superior potential for immediate breakout.

See these time-sensitive tickers now >>

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Want the latest recommendations from Zacks Investment Research? Today, you can download 7 Best Stocks for the Next 30 Days. Click to get this free reportMeridian Bioscience Inc. (VIVO) : Free Stock Analysis ReportCRISPR Therapeutics AG (CRSP) : Free Stock Analysis ReportTo read this article on Zacks.com click here.

Read more:
Meridian Bioscience, CRISPR Therapeutics, Tilray and Uber highlighted as Zacks Bull and Bear of the Day - Yahoo Finance

Recommendation and review posted by Bethany Smith

The Global CRISPR Technology Market was valued at USD 696.7 Million in 2020 and is forecasted to reach USD 3.94 Bn at a CAGR of +24% by 2028.

CRISPR is a technology that can be used to edit genes and, as such, will likely change the world. The essence of CRISPR is simple: its a way of finding a specific bit of DNA inside a cell. After that, the next step in CRISPR gene editing is usually to alter that piece of DNA.

CRISPR-Cas9 was adapted from a naturally occurring genome editing system in bacteria. The bacteria capture snippets of DNA from invading viruses and use them to create DNA segments known as CRISPR arrays. The CRISPR arrays allow the bacteria to remember the viruses (or closely related ones).

With CRISPR, scientists can create a short RNA template in just a few days using free software and a DNA starter kit that costs $65 plus shipping. Unlike protein-based technologies, the RNA in CRISPR can be reprogrammed to target multiple genes.

The company has a healthy balance sheet with $1.4 billion in cash and very little debt. CRISPR has the financial flexibility to fund its research programs for many more years to come. So it can prove to be an attractive investment for healthcare investors in the next five years.

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Major Players Covered in this Report:

CRISPR Technology Market Study assures you to advise higher than your competition. With Structured tables and figures examining the CRISPR Technology, the research document provides you a leading product, submarkets, revenue size and forecast to 2028.

The study report offers a comprehensive analysis of CRISPR Technology market size across the globe as regional and country level market size analysis, CAGR estimation of market growth during the forecast period, revenue, key drivers, competitive background and sales analysis of the payers. Along with that, the report explains the major challenges and risks to face in the forecast period.

The research report of the CRISPR Technology market offers broad analysis about the industry on the basis of different key segments. Moreover, the research report presents a comprehensive analysis about the opportunities, new products, and technological innovations in the market for the players.

Additionally, the research report on CRISPR Technology market provides an in depth analysis about market status, market size, revenue share, industry development trends, products advantages and disadvantages of the enterprise, enterprise competition pattern, industrial policy and regional industrial layout characteristics. Thus the study report offers a comprehensive analysis of market size across the globe as regional and country level market size analysis, estimation of market growth during the forecast period.

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Market segmentation by Product Type:

Market segmentation by Applications:

Market segmentation by regions:

This study also covers company profiling, specifications and product picture, sales, market share and contact information of various regional, international and local vendors of Global CRISPR Technology Market. The market opposition is frequently developing greater with the rise in scientific innovation and M&A activities in the industry. Additionally, many local and regional vendors are offering specific application products for varied end-users. The new merchant applicants in the market are finding it hard to compete with the international vendors based on reliability, quality and modernism in technology.

Detailed TOC of CRISPR Technology Market Research Report-

CRISPR Technology Introduction and Market Overview

CRISPR Technology Market, by Application

CRISPR Technology Industry Chain Analysis

CRISPR Technology Market, by Type

Industry Manufacture, Consumption, Export, Import by Regions

Industry Value ($) by Region

CRISPR Technology Market Status and SWOT Analysis by Regions

Major Region of CRISPR Technology Market

Major Companies List

Conclusion

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CRISPR Technology Market hits at $3.94 Bn by 2028 with Thermo Fisher Scientific, Merck KGaA, GenScript, Integrated DNA Technologies (IDT) - The...

Recommendation and review posted by Bethany Smith

California headquartered, Caribou, isa clinical-stage CRISPR genome editing biotechnology company.

The collaboration leverages Caribous next-generation CRISPR genome editing technology platform and AbbVies antigen-specific binders. AbbVie will utilize Caribous next-generation Cas12a CRISPR hybrid RNA-DNA (chRDNA) genome editing and cell therapy technologies to research and develop two new CAR-T cell therapies.

Under the terms of deal, Caribou will receive US$40m in an upfront cash payment and equity investment and up to US$300m in future development, regulatory, and launch milestones.

Although allogeneic CAR-T cell therapies have shown early promise in some cancer patients, the need for overcoming the rejection of allogeneic CAR-T cells by the host immune system remains a key challenge to their broader development, said the companies.

Employing Caribous CRISPR genome editing platform to engineer CAR-T cells to withstand host immune attack would enable the development of the next-generation cellular therapies to benefit a broader patient population, claim the partners.

Caribou will conduct certain pre-clinical research, development, and manufacturing activities for the collaboration programs, and AbbVie will reimburse Caribou for all such activities pursuant to the collaboration, while AbbVie will be responsible for all clinical development, commercialization, and manufacturing efforts.

AbbVie also has the option to pay a fee to expand the collaboration to include up to an additional two CAR-T cell therapies.

We are excited to partner with AbbVie on the development of new CAR-T cell therapies. This collaboration validates Caribous differentiated next-generation CRISPR genome editing technologies that provide best-in-class efficiency and specificity, said Rachel Haurwitz, CEO of Caribou.

CAR-T therapies have shown to be a promising breakthrough in cancer treatment, Collaborating with Caribou and their cutting-edge CRISPR platform will help AbbVie advance our efforts to deliver new hope for patients, said Steve Davidsen, VP, oncology discovery, AbbVie.

Outside of this collaboration, Caribou is advancing an internal pipeline of allogeneic cell therapies for oncology. CB-010, its lead allogeneic CAR-T cell program, targets CD19 and is being evaluated in a Phase 1 clinical trial for patients with relapsed/refractory B cell non-Hodgkin lymphoma.

CB-011, Caribous second allogeneic CAR-T cell therapy, targets BCMA for multiple myeloma, while CB-012, Caribous third allogeneic CAR-T cell therapy, targets CD371 for acute myeloid leukemia.

CB-011 and CB-012 are in preclinical development.

Additionally, Caribou is developing iPSC-derived allogeneic natural killer (NK) cell therapies for solid tumors.

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AbbVie and Caribou partner on CAR-T cell therapy development - BioPharma-Reporter.com

Recommendation and review posted by Bethany Smith

Due to the unique consequences of the pandemic, we are able to catch a glimpse of a potential future. One where we sit, plugged into our computers, devoid of physical human connection. What will society look like after the pandemic? Will we continue to stay isolated? Surely advancements in technology have played key roles in prolonging our lives, allowing us to continue to work and socialise, but to what end? With these newly emerging technologies are we destined to live forever, in a suspended state, in front of the glow of our 4k computer screens? Will gene editing technologies be used to keep us alive forever so that we can binge watch infinite Netflix shows, send meaningless emails and scroll through social media feeds?

Q4 2020 hedge fund letters, conferences and more

The Institute of Zoology of the China Academy of Science has successfully prolonged the lives of mice by using CRISPR/Cas 9. CRISPR has become a relatively simple and popular way to edit strands of DNA. The CRISPR/Cas 9 study found a gene tied to cellular senescence (which tells cells to stop growing) and also, that CRISPR/Cas9 treatment can make partially dormant the aging process. CRISPR/Cas9 treatment allowed mice to live 25% longer and be physically stronger. Biologists see these results being relatively easy to reproduce on humans in a clinical setting.

Company managements looking to achieve earnings growth often default to cost cutting, stock buyback, accounting gimmicks and other methods. But there is another way. More often than not, managements overlook pricing as a driver of earnings growth. Pricing power can be an effect way of boosting a company's bottom line. Read More

Existing in a world where individuals are able to receive treatment to live longer borders dystopian science fiction. The treatment can reduce the need for medical attention by potentially reducing injuries, heart attacks, and organ failures.

In part, due to breakthroughs in the tech and science industries, life expectancy in the 21st century is projected to steadily increase. In a study published in the Lancet, average life expectancy is predicted to rise in 35 industrialized countries by 4.4 years in men and women by 2030.

Life expectancy will likely increase as we migrate away from laborer positions. Currently, the National Center for Health Statistics puts unintentional accidents, primarily happening within labor positions, as the third leading cause of death in the U.S. Many industrialized countries, like the U.S., have been witness to a slow disappearance of the labor class, but the pandemic has made that increasingly more apparent. Businesses are shifting towards automated technology to replace physical human interactions to curb the spread of Covid-19. Even within agriculture and farming industries, already abundant with machine automation, companies are pushing even further away from human labor in an effort to reduce virus rates.

Simultaneously, we are witness to the emergence of new and remote jobs and work settings. Homes are new sites for schooling, work and entertainment. Before the pandemic there was already a struggle to maintain a separation between home and work identities. The pandemic has exacerbated this problem. How do we find rest and recuperation when we are living within the office space? Time on the job stretches on forever as we receive work emails while watching Netflix with our families. If life expectancy in humans gets extended by using CRISPR/Cas 9 are we just creating our own version of purgatory? Is our future one where our time working stretches on seemingly infinitely while we simultaneously cease to age? Are we becoming the perfect machine, one that is held together by technological advancements that inadvertently disembody and dehumanise us?

Was society slowly transitioning toward isolation before the pandemic? Is isolation a byproduct of neoliberalism? Gated communities, mass incarceration, office cubicles, segregation in neighborhoods, retirement homes, hospitals, national borders and private properties all verify how neoliberalism operates. Now we are being asked to isolate within the confines of our homes. When the pandemic is over, will people continue to order food, work, shop and socialise from isolated and often virtual spaces or will we be able to shift back into the more public and physical? Will the last remaining physical laborers, those that are delivering goods to doorsteps, be replaced by driverless vehicles?

About the Author

Matthew Bacher is an author and professional artist and is working towards his MFA in Painting and Printmaking at San Diego State University. His work deals with postmodern ideas around nature, technology and the self. You can find his artwork atmbacherart.com.

More:
CRISPR Treatment Offers The Potential To Live Forever - ValueWalk

Recommendation and review posted by Bethany Smith

The term genome generally refers to the entire sequence of DNA of an organism. The genome includes genes: sequences of DNA with specific functions that are involved in the production of the proteins needed to carry out many biological roles. Genome editing is the deliberate alteration of a selected DNA sequence in a living cell. A strand of DNA is cut at a specific point and naturally existing cellular repair mechanisms, then fix the broken DNA strands.

The Australia Gene Editing market 2021 research provides a basic overview of the industry including definitions, classifications, applications and industry chain structure. The Australia Gene Editing market analysis is provided for the international markets including development trends, competitive landscape analysis, and key regions development status. Development policies and plans are discussed as well as manufacturing processes and cost structures are also analyzed. This report also states import/export consumption, supply and demand Figures, cost, price, revenue and gross margins.

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Top Vendors of Australia Gene Editing Market:-

CRISPR Therapeutics, Thermo Fisher Scientific, GenScript Corporation, Merck KgaA, Sangamo Therapeutics, Inc., Horizon Discovery Group, Integrated DNA Technologies, New England Biolabs, OriGene Technologies, Lonza Group, and Editas Medicine.

Australia Gene Editing Market, By Application

Australia Gene Editing Market, By Technology

Australia Gene Editing Market, By End-user:

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The study is a source of reliable data on:

-Key market segments and sub-segments

-Evolving market trends and dynamics

-Changing supply and demand scenarios

-Quantifying market opportunities through market sizing and market forecasting

-Tracking current trends/opportunities/challenges

-Competitive insights

-Opportunity mapping in terms of technological breakthroughs

The Australia Gene Editing market research report completely covers the vital statistics of the capacity, Development, value, cost/profit, supply/demand import/export, further divided by company and country, and by application/type for best possible updated data representation in the figures, tables, pie chart, and graphs.

Fundamentals of Table of Content:

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Australia Gene Editing Market to Garner Astonishing CAGR by 2028 with Top Key Players: CRISPR Therapeutics, Thermo Fisher Scientific, GenScript...

Recommendation and review posted by Bethany Smith

The newresearchusesdatafroma 2018study,whichis stillthe largestanalysisof ancient DNAfrom Britain ever conducted,thatidentifieda >90% replacement of the genetic ancestry of people living in Britain between 2500-2100 BC. Thiscoincidedwith the introduction of Beakermaterial culture and burialpractices,andwas interpreted to indicatethat there were substantial movements of people into Britain from continental Europe during this period.

At the time it was published,popular coverage of the original studyspeculatedthat this wasa rapidevent, potentially involving invasionby male warriors, butthe new study has foundfromdetailed analysis that it was more likely a long-term process, taking place over maybe 10-16 generations,withboth men and women moving for a variety of reasons thatmight haveincluded exchange, pilgrimage, and the pasturing of animals.Incoming populationsand their descendants tended to bury their dead, but local groups probably continued to cremate their dead, which destroys the DNA, or treatthemin ways which leavenorecord.Thearchaeologically invisiblelocalpopulationare only seenwhen they have children with groups who buried their dead. Thismay be partly responsible for why this change in ancestryappearedso rapidat first.

Dr Tom Booth, archaeologist at The Francis Crick Institute has said: Initially it looks like groups of locals and incomers and their descendants lived in parallel with one anotherto some extent occupying the same landscapes, slowly integrating and only having children with each other infrequently.After around 300 yearsthey start having children together more liberally itsat this point, the older population then have a much lower genetic legacyoverall.Why they have such a small overall genetic legacy is still a mystery. It could be thattherejust werent so many people living in Britain at the time these Beaker groups move in from continental Europe.

ProfIanBarnes, Researcher and Division Lead at the Natural History Museum has said:Anissue we face is that wealsodont know how many people there were from either group,althoughpopulation size may be declining inthe localpopulation of Britain at the end of Neolithic. It may be that the reason whywe seem to pick up so many genetic relatives in the Bronze Ageis becauseonly small groups of people were moving into Britain.

The new study also highlights how genetic ties were referenced variably in death among burials in the Stonehenge landscape.A man and his juvenile son were buried next to one another in a cemetery on Amesbury Down.By contrast, aman, hisnephewand his nephews daughter were buried across three different cemeteries separated by severalkilometres.A young man was buried on Boscombe Down with the skull of his paternal cousin or half-brother at his feet.

Prof. JoannaBrck, archaeologist at University College Dublin, said: Existing interpretations of the genetic evidence paint a picture of a patriarchal society, in which male immigrants married localwomen. Our research shows that although links with paternal relatives were important, kinship organization was variable, and other relationships, including with maternal kin, were also significant.Sometimes, people who were not genetically related to each other could also be viewed as kin.

The study found that it is likely there was cultural exchange between existing local groups and incomers.Dr Booth continues Even though they have no ancestry from the older population, they incorporatetheir monuments into their belief systems very quickly. They are burying people in these areas to reference these monuments as prestigious areas to bury their dead even though itwasnttheir genetic ancestors who built them.Stonehengeand its surrounding landscapeareemblematic to a certain extent because its important toallgroups in this period and when theyintegrate,it maintains its importance.

The paper was published inCambridge Archaeological Journal on 11 February 2021.

Notes foreditors

Media contact: Tel: +44 (0)779 969 0151 Email:press@nhm.ac.uk

About the Natural History Museum:

The Natural History Museum is both a world-leading science researchcentreand the most-visited natural history museum in Europe. With a vision of a future in which both people and the planet thrive, it is uniquely positioned to be a powerful champion for balancing humanitys needs with those of the natural world.

It is custodian of one of the worlds most important scientific collections comprising over 80 million specimens. The scale of this collection enables researchers from all over the world to document how species have and continue to respond to environmental changes - which is vital in helping predict what might happen in the future and informing future policies and plans to help the planet.

The Museums 300 scientists continue to represent one of the largest groups in the world studying and enabling research into every aspect of the natural world. Their science is contributing critical data to help the global fight to save the future of the planet from the major threats of climate change and biodiversity loss through to finding solutions such as the sustainable extraction of natural resources.

The Museum uses its enormous global reach and influence to meet its mission to create advocates for the planet - to inform, inspire and empower everyone to make a difference for nature. We welcome over five million visitors each year; our digital output reaches hundreds of thousands of people in over 200 countries each month and our touring exhibitions have been seen by around 30 million people in the last 10 years.

The Francis Crick Instituteis a biomedical discovery institute dedicated to understanding the fundamental biology underlying health and disease. Its work is helping to understand why disease develops and to translate discoveries into new ways to prevent, diagnose and treat illnesses such as cancer, heart disease, stroke, infections, and neurodegenerative diseases.

An independentorganisation, its founding partners are the Medical Research Council (MRC), Cancer Research UK,Wellcome, UCL (University College London), Imperial College London and Kings College London.

The Crick was formed in 2015, and in 2016 it moved into a brand new state-of-the-art building in central London which brings together 1500 scientists and support staff working collaboratively across disciplines, making it the biggest biomedical research facility under a single roof in Europe.http://crick.ac.uk/

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Experts discover new information on cultural exchange between different genetic groups and burials of close genetic relatives in the Stonehenge...

Recommendation and review posted by Bethany Smith

Sperm are simple cells with a straightforward job: swim until they reach an egg, then fertilize it. But in mice, some sperm resort to divisive tactics in order to gain the advantage.

A study published on February 4 in the journal PLOS Genetics shows that a genetic variation in mouse sperm, called the t-type, can give a swimmer the upper hand. These t-type sperm are able to spread a protein called RAC1 that essentially poisons other sperm. T-type sperm plant the seeds of destruction early in their development, then fortify themselves against RAC-1, Brandon Specktor reports for Live Science. When it comes time to race for the egg, the t-type sperm can swim in a straight line while poisoned sperm swim in hapless circles until they die.

We found out that the level of this protein can be more or less active, depending on whether the sperm have the gene to make it, and whether that gene is flipped on like a light switch, says biologist Alexandra Amaral of the Max Planck Institute for Molecular Genetics to Kassidy Vavra at Inverse. The level of protein that is on has to be quite well regulated. If it is too much, sperm don't move well. And if its too low, it also doesnt move well theyre kind of in circles.

T-type sperm produce the RAC1 protein at full throttle.

If all of the sperm in a group are t-type, and theyre all making RAC1, they will all struggle because there is so much of the poisonous protein going around, Sara Rigby reports for Science Focus magazine. On the other hand, if there are no t-type sperm present, then all the other sperm remain relatively healthy and swim well because theres no overabundance of RAC1. However, if a cohort has a mix of t-type and normal sperm, then t-type will have the advantage.

"The trick is that the t-haplotype 'poisons' all sperm, but at the same time produces an antidote, which acts only in t-sperm and protects them," says Bernhard Herrmann, director of the Max Planck Institute for Molecular Genetics, in a statement. "Imagine a marathon, in which all participants get poisoned drinking water, but some runners also take an antidote."

The t-type sperm do the equivalent of poisoning the drinking water early in sperm development, affecting both themselves and their non-variant peers. All of the sperm inherit genes that make it difficult to interpret the chemical signals around them. But in the final cell division of sperm development, when half of a cells genes go to one sperm and the other half to another, only the sperm that inherit the t-type variation have an extra set of genes that reverses the poisons effect, per Live Science.

The poisoned sperm end up swimming in circles, unable to advance in their quest. But the impervious t-type sperm swim ahead. In this case, theres a 99 percent chance that the sperm that fertilizes the egg first will have the t-type variation. The research shows the importance of small genetic variations in sperms success, Amaral tells Inverse.

The study was conducted in about 100 mouse sperm cells, but not all species sperm behave the same way, University of California, Berkeley, cell biologist Polina Lishko tells Inverse. The study is preliminary, but future research could illuminate the specific molecular mechanism behind RAC1 that makes it damaging to sperm at high levels.

An earlier study showed a similar effect of RAC1 on bull sperm, which is more similar to human sperm than a mouses is. Amaral says that the team plans to conduct future research with human sperm, to see if RAC1 might be involved with some cases of male infertility.

Go here to see the original:
Mice Sperm Sabotage Other Swimmers With Poison | Smart News - Smithsonian Magazine

Recommendation and review posted by Bethany Smith

It is not just a mother's love that endures.

A woman's influence on her children's health persists well into late middle age, according to a study published in the November issue of the European Journal of Preventive Cardiology.

The study, which enrolled almost 2,000 men and followed their families over 46 years - from 1971 to 2017 - found that the sons of women with heart-healthy lifestyles live nearly a decade longer without developing cardiovascular disease than those whose mothers have unhealthy lifestyles.

The study looked at the influence of both parents on offspring but found that men had little influence on their children's heart health in later life apart from the genes they passed on to them.

According to Dr Rohit Khurana, senior consultant cardiologist with The Harley Street Heart and Vascular Centre at Mount Elizabeth Medical Centre, told The New Paper: "While a pregnant woman's cardiovascular health (CVH) and lifestyle choices during pregnancy can affect her offspring's CVH, long-term CVH is also affected through parental behaviours and environmental influences.

"Children observe and acquire health behaviours within the family environment, with role modelling by primary caregivers being a significant contributor to children's long-term lifestyle choices.

"If primary caregivers, usually mothers, practise and instil a healthy lifestyle of balanced diets, regular exercise and minimal to zero alcohol and tobacco intake... their children are likely to continue those behaviours in adulthood.

"Those children will then pass good habits down to their children, thereby decreasing the risk of CVD (cardiovascular disease) within families for generations."

He added: "The research shows that parents, particularly mothers, are the gatekeepers of their children's CVH during formative years, which influences adult life. Numerous studies have proved that good lifestyle choices are a greater determining factor compared with genetics when it comes to optimal CVH."

According to the study, CVD incidence rates were higher among sons than daughters, (with one reason being that) women are less likely to indulge in risky behaviours.

For example, according to the World Health Organisation, about 40 per cent of the world's male population smokes, compared with only 9 per cent of women, and men are almost twice as likely to binge drink as women.

Studies also show that women generally eat more healthily and consume more fruit and vegetables than men.

Finally, women develop CVD later in life, after menopause and typically in their 60s or older; so fewer of the female participants in the study had reached the age when we would expect to see signs of CVD.

Mothers are still the primary caretakers of young ones, with more direct daily influence on diet and behaviour than fathers. They are still more likely to be disciplining, providing emotional support and generally monitoring the daily activities of their children.

Taking good care of your CVH during pregnancy is important because the heart works harder by increasing the body's blood volume to support a growing baby.

If you are a diabetic, a smoker or have high blood pressure during pregnancy, each of these things makes it harder for your heart to pump extra blood throughout your circulatory system, increasing the likelihood of maternal and neonatal mortality and morbidity.

This does not mean women with CVD risk factors should not get pregnant - it just means they should practise good preconception heart care including smoking cessation, cutting back on alcohol and weight management through regular exercise and a healthy diet rich in fruit, vegetables and fibre.

After women give birth, they should continue practising healthy behaviours at home as good examples to help children make positive choices, which become lifelong habits.

Telling children what to do will not always work as they need to see parents walk the talk.

Read more:
Sons of heart-healthy mums more likely to live 10 years longer: Study - The New Paper

Recommendation and review posted by Bethany Smith

Full Guideline: Hormonal Replacement in Hypopituitarism in AdultsJCEM | October 2016Maria Fleseriu (Chair), Ibrahim A. Hashim, Niki Karavitaki, Shlomo Melmed, M. Hassan Murad, Roberto Salvatori, and Mary H. Samuels

The 2016 guideline addresses:

The guideline addresses special circumstances that may affect the treatment of patients with hypopituitarism, including pregnancy care, post-surgical care following pituitary or other operations, treatment in combination with anti-epilepsy medication, and care following pituitary apoplexya serious condition that occurs when there is bleeding into the gland or blood flow to it is blocked.

Recommendations from the guideline include:

+ 1.0 Diagnosis of hypopituitarism

Central adrenal insufficiency

Central hypothyroidism

GH deficiency

Central hypogonadism in males

Central hypogonadism in females

Central diabetes insipidus

+ 2.0 Treatment

Hormonal replacement in panhypopituitarism

Glucocorticoid replacement

Adrenal crisis

Thyroid hormone replacement

Testosterone replacement

Estrogen replacement in premenopausal women

GH replacement therapy

Diabetes insipidus

Interactions between replacement hormones

Glucocorticoids and GH

Glucocorticoids and thyroid hormone

Glucocorticoids and estrogen

GH and thyroid hormones

Estrogen and thyroid hormones

GH and estrogen

Glucocorticoids and diabetes insipidus

Risk of hormonal over-replacement in hypopituitarism

Bone disease

Cardiovascular risks in patients with hypopituitarism on replacement therapy

Glucocorticoid over-replacement

Thyroid replacement

+ 3.0 Special circumstances

Cushings disease

Prolactinomas

GH replacement in cured acromegaly after surgery and/or radiation

Perioperative management of hypopituitarism

Pituitary surgery

Non-pituitary surgery

Management of hypopituitarism in pregnancy

Glucocorticoids

Thyroid

Desmopressin

Growth hormone

Management of hypopituitarism in pituitary apoplexy

Treatment of hypopituitarism in patients receiving antiepileptic medications

Read more from the original source:
Hormone Replacement in Hypopituitarism Guideline Resources ...

Recommendation and review posted by Bethany Smith

A gene therapy costing 16 crore is the only shot of life for nearly 200 children with Spinal Muscular Atrophy (SMA) Type 1, a rare genetic disease, in Karnataka.

Last month, the therapy Zolgensma was offered free to a 14-month-old baby from Bhatkal (Uttara Kannada) who was the lucky winner of a lottery through a compassionate access programme by Novartis, the Swiss drugmaker. This lottery is held once in two weeks for SMA children across the world and doctors at Baptist Hospital, that has a dedicated Paediatric Neuromuscular Service, are hoping more children will benefit.

The therapy is a one-time infusion that takes about an hour, Ann Agnes Mathew, Consultant Paediatric Neurologist and Neuromascular Specialist, at Baptist Hospital told The Hindu. The therapy was approved by U.S. regulators in May 2019 and has since then turned into a miracle drug for this rare disorder that destroys a babys muscle control.

SMA is a disease caused by loss of nerve cells, which carry electrical signals from the brain to the muscles. The protein needed for this signalling is coded by a gene for which everyone has two copies - one from the mother and the other from the father. A child develops this disorder only if both the copies are faulty. Without treatment, this disease is ultimately fatal, said Dr. Mathew. The disease as it progresses, makes it extremely difficult for the babies to carry out basic activities like sitting up, lifting their head or swallowing milk.

Pointing out that the current treatment options range from medicines, which increase these proteins, to replacing the faulty gene, the doctor said, Zolgensma is a revolutionary treatment, which works by supplying a healthy copy of the faulty gene, which allows nerve cells to then start producing the needed protein. That halts deterioration of the nerve cells and allows the baby to develop more normally.

The drug has a 14-day shelf life and when it was sent from U.S. for the Bhatkal baby, it was stuck with customs for three days in mid-January making doctors jittery. Dr. Mathew said she had to personally meet the Customs officials to get it released. When we explained the situation to them, they immediately released it. Any further delay would have been risky. The parents have taken a house on rent and are staying near the hospital for follow up. The baby is doing fine now, she said.

Pointing out that 38 babies had succumbed to the rare disease in Karnataka in over one-and-a-half years, Dr. Mathew said most families have given up hope as they cannot afford the treatment.

The Paediatric Neuromuscular Service at Baptist Hospital is a pioneering centre in the country with a multidisciplinary team of a paediatric neurologist, paediatric neuromuscular specialist, paediatric geneticist, paediatric pulmonologist, paediatric intensivist, paediatric cardiologist and paediatric endocrinologist providing comprehensive care under one roof. This service is run in collaboration with Organisation for Rare Diseases India, a NGO.

A Bengaluru-based couple - Naveen Kumar and Jyothi - have taken to crowdfunding on ImpactGuru.com, a crowdfunding platform, to cover the cost of Zolgensma therapy for their 10-month-old baby Janish who was diagnosed with SMA.

Mr. Kumar, who works as an insurance surveyor and barely earns 30,000 a month, cannot afford the expensive treatment.

The couple were counting their babys milestones after his birth in February 2020. They caught his first smile and his first laugh but baby Janish never went past his first two milestones. The parents then rushed him to a pediatrician and from there the baby was referred to Baptist Hospital, said Dr. Ann Agnes Mathew, who has been treating the baby for the last five months.

Piyush Jain, co-founder and CEO, ImpactGuru.com, said over 22 lakh has been raised so far for baby Janish from over 1,500 donors.

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16 crore drug is the hope for SMA patients - The Hindu

Recommendation and review posted by Bethany Smith

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Edinburg, TX Can a medical tool predict the risk of certain medications based on a patients genetics, and, if so, could this information lead to doctors customizing a patients drug therapy? The tool being studied at DHR Health Institute for Research & Development to answer these questions is Cipheromes Xentinel Lighthouse, and this pilot study will also evaluate the resulting savings in health-care costs.

After a stroke or heart attack, the standard treatment is Percutaneous Coronary Intervention (PCI), or stent placement. A stent opens blood vessels in the heart that have narrowed due to the buildup of plaque. Following PCI, doctors typically prescribe aspirin and a P2Y12 inhibitor, such as clopidogrel (Plavix), to reduce the risk of stent thrombosis, which is the formation of a blood clot on the surface of the stent.

Lighthouse is a support tool that allows physicians to predict how a patient will respond to drug therapies based on that patients genetic makeup. Cipherome, Inc. chose South Texas for this pilot project to better understand how the genetic differences in Hispanic/Latino patients relate to the effectiveness and safety of medications used to treat them.

Most prior studies to anticipate major bleeding caused by this drug therapy have been based on data collected from Caucasian patients, have not been based on apatients individual risk profile, and have not been strong predictors of thrombosis risk, says Dr. Herschl Silberman, a cardiologist at DHR Health and a Clinical Research Scientist, DHR Health Institute for Research & Development. This lack of effective clinical tools has resulted in high numbers of emergency room visits and patients being readmitted to the hospital with stent thrombosis, bleeding, and other complications.

In 2019 alone, over 1.5 million patients visited an emergency room or were hospitalized due to unexpected reactions to drug therapies, and 174,000 people in the U.S. lost their lives due to these adverse reactions. Recent studies show the possibility of an 80-percent reduction in PCI 90-day complications by using a patients personalized genetic information.

Personalized medicine brings great promise to improve outcomes for our patients and we are focused on pioneering innovative practical solutions that can produce better results for our clinicians and patients, says Dr. Sohail Rao, CEO of DHR Health Institute of Research and Development (DHR IRD). DHR IRDs credibility and service to the large Hispanic population in South Texas is the ideal place for our first deployment of clinical decision support systems, says Ilsong Lee, Cipherome CEO.

This study is open to patients 18 years of age and older who have been prescribed clopidogrel, prasugrel (Effient), or ticagrelor (Brilinta) after a PCI with at least one stent placement. There are two study arms, or groups, one receiving clopidogrel but no genotyping to identify the patients genetic makeup and the other receiving genotype-guided therapy. Patients in the study will be evaluated for a period of one year.

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The First Gene Based Project to be conducted in the Rio Grande Valley - Mega Doctor News

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Spondyloarthritis is an umbrella term that describes different types of arthritis. These types mainly affect the spine, but they can also cause symptoms in other parts of the body.

There has been some debate about whether spondyloarthritis is an autoimmune condition or an autoinflammatory condition. However, recent research suggests that spondyloarthritis is indeed an autoimmune condition.

This article will explore spondyloarthritis in detail. Specifically, it will examine the different types, symptoms, and treatment options associated with the condition.

There are several subtypes of spondyloarthritis. Each can involve a different part of the body.

The following sections will look at some of these types in more detail.

Ankylosing spondylitis is the most common form of spondyloarthritis. It involves ligaments, tendons, and joint capsules attaching to bones in the spine and peripheral joints.

It can cause the bones in the spine to fuse together, leading to stiffness and immobility.

Learn more about ankylosing spondylitis here.

This type of spondyloarthritis primarily involves the joints in the spine and pelvis.

Axial spondyloarthritis causes back pain and affects around 5.5 million people in the United States.

Like axial spondyloarthritis, the non-radiographic form also affects the spine and causes lower back pain.

However, the effects of non-radiographic spondyloarthritis are not visible on X-rays. They are only visible on more sensitive imaging tests, such as MRI scans.

Peripheral spondyloarthritis describes a number of spondyloarthritis subsets.

It mainly affects the hands and feet. However, it can also cause inflammation in the:

Both rheumatoid arthritis and spondyloarthritis are very common. Although they share some similarities, the conditions also have significant differences.

Spondyloarthritis tends to be more common in males, whereas rheumatoid arthritis is more common in females.

Rheumatoid arthritis symptoms typically start appearing when a person is around 4050 years of age. The symptoms of spondyloarthritis usually occur earlier than this.

The early symptoms of rheumatoid arthritis usually affect the hands and feet. The early symptoms of spondyloarthritis usually start with back pain.

Learn more about the early signs and symptoms of rheumatoid arthritis here.

People often develop spondyloarthritis in their teenage years or 20s. Those with the following characteristics may be more likely to experience spondyloarthritis:

It is important to note that spondyloarthritis is notoriously difficult to diagnose in females. This could mean that it is more common in females than some statistics may show.

Lower back or hip pain is a common early symptom. However, symptoms can vary depending on the type of spondyloarthritis a person has.

Inflammation elsewhere in the body is a symptom of spondyloarthritis. It can especially affect the:

Spondyloarthritis-related inflammation can cause:

Another symptom of spondyloarthritis and the swelling it causes is psoriatic rashes. These may appear differently depending on a persons skin color.

Learn more about psoriasis on black skin here.

These symptoms may be particularly painful first thing in the morning or after periods of rest.

Untreated spondyloarthritis could lead to a person developing the following conditions:

A person who is experiencing symptoms of spondyloarthritis should contact a doctor to treat the condition. This may help prevent these complications.

If a person has had chronic lower back pain since before the age of 40 years, they may have spondyloarthritis. People often assume that they simply have back pain due to poor posture or other mechanical issues.

Because the pain can come and go, some people may assume that the pain is not important. However, not seeking treatment for spondyloarthritis can lead to complications later on.

A person who suspects that they have spondyloarthritis should contact a doctor. They should provide the doctor with details about their pain onset and whether or not they have other inflammatory symptoms that might suggest the presence of spondyloarthritis.

A doctor will diagnose spondyloarthritis by taking a persons medical history and performing a physical exam.

Imaging can help confirm a diagnosis. The doctor may request an MRI scan if an X-ray does not show damage but a person has symptoms that suggest the presence of spondyloarthritis.

A blood test is also available for the HLA-B27 gene, which is a gene associated with the condition. However, testing positive for the gene does not necessarily mean that a person has spondyloarthritis.

The doctor can also perform ESR tests or CRP tests on the blood in order to determine if swelling is present in the body. This can also help diagnose spondyloarthritis.

The doctor may also choose to carry out a complete blood count, to diagnose anemia, or a metabolic panel, to analyze a persons kidney and liver function.

Sometimes, medical professionals can mistake spondyloarthritis for other similar conditions, which can delay diagnosis and treatment. This is especially the case among females.

There is currently no cure for this condition. However, treatment can help relieve the symptoms and slow the progress of the condition.

Some treatment options include:

Biologics are very effective but expensive. These drugs can also increase a persons risk of infection.

Also, physical therapy can help restore range of motion in the affected joints.

One 2020 study involved putting 100 people with axial spondyloarthritis, non-radiographic axial spondyloarthritis, or psoriatic arthritis with axial involvement on physical therapy treatment programs.

The therapy significantly improved the pain that the condition caused, including among those with secondary conditions such as fibromyalgia.

Occupational therapy can also help a person improve or maintain their ability to perform day-to-day activities. An occupational therapist can provide recommendations and assistive devices to help prevent further injury.

Living with spondyloarthritis can make performing certain everyday tasks more difficult, but it is possible to manage the symptoms. Also, the condition does not usually affect a persons life expectancy significantly.

Symptoms such as pain and fatigue may come and go, and treatments can help a person live with this condition.

Some behavioral changes can also make living with spondyloarthritis easier. These changes include:

There are also spondyloarthritis support groups available for people who may need additional support.

Not seeking treatment for spondyloarthritis can lead to complications. Joints can fuse, for example, which may cause severe stiffness or immobility.

The symptoms of spondyloarthritis can come and go. However, even if a person does not constantly experience symptoms, they should still contact a doctor.

Not seeking treatment can lead to more complications of the condition. For example, it could become increasingly painful.

With the right treatment, people with spondyloarthritis can live an active life. Although there is no way to cure the condition, it is possible to manage the symptoms and prevent disease progression.

Making certain behavioral changes and trying medical treatments can make the pain and inflammation of spondyloarthritis more manageable.

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Spondyloarthritis: Types, symptoms, treatment, and more - Medical News Today

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Even studies have presented that those children, who were born out of hypothyroidism mothers during pregnancy, have lower IQ and impaired psycho-motor (mental and motor) development. But, fret not! If the condition is properly controlled and treated well, then those women with hypothyroidism can also have healthier babies.

Since the thyroid gland of the unborn child takes time to function on its own, its completely dependent on the mother for the thyroid hormones. Development and functioning of babys thyroid gland do not take place until about the end of the first trimester of pregnancy. Therefore, its suggested that women should start getting their thyroid disorder managed before conceiving so that impaired neurological functioning, stunted growth and physical deformities in the children can be avoided.

There is always best to plan for pregnancy and to consult with your physician to ensure your thyroid status and treatment are optimised prior to becoming pregnant and monitored throughout your pregnancy. However, if this does not happen and you find out you are pregnant, you should contact your physician immediately to arrange for increased testing of your thyroid functions and a potential change in your medication.

Untreated or poorly controlled hypothyroidism can also lead to:- Miscarriage- Premature birth- Pre-eclampsia - StillbirthSo, it is vital for pregnant women with hypothyroidism to take the recommended thyroid medication consistently.

How Is Hypothyroidism Treated During Pregnancy? The treatment of hypothyroidism in pregnant women is similar to that of the regular treatment. Doctors recommend synthetic T4 so that it compensates the presence of essential hormones in the body. The medication should be taken regularly so that a steady blood level of thyroid hormone gets adjusted within the normal range as the requirement of thyroid hormones increases during pregnancy. Therefore, it is a routine practice to monitor the blood level of the thyroid stimulating hormone (TSH) during pregnancy. Hyperthyroidism women can have healthy pregnancy by getting early prenatal care and working with their healthcare providers in the management of their disease.

Also read: Expert Speak: How To Lower The Risk Of Neural Birth Defects

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Expert Speak: Hypothyroidism And Its Effect On An Unborn Child - Femina

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The ongoing trial is enrolling patients at 62 locations in the United States and worldwide.

Neeraj Agarwal, MD, discusses the rationale for the ongoing phase 3 randomized, open-label, CONTACT-02 study (NCT04446117) of the multikinase inhibitor cabozantinib (Cabometyx) plus the PD-L1 inhibitor atezolizumab (Tecentriq) versus second novel hormone therapy (NHT; abiraterone acetate [Zytiga] or enzalutamide [Xtandi], etc) in patients with metastatic, castration-resistant prostate cancer (mCRPC) who previously received 1 NHT to treat metastatic castration-sensitive prostate cancer, non-metastatic CRPC, or mCRPC.

Primary outcomes measures are overall survival and progression-free survival, with objective response rate as a secondary outcome measure. The target enrollment is 580 patients, and there are 62 study locations for the trial in the United States and worldwide.

Agarwal is a professor of Medicine, physician and investigator at the Huntsman Cancer Institute, University of Utah. He directs the Genitourinary Oncology Program and the Center of Investigational Therapeutics, and co-leads the Experimental Therapeutic Program.

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Dr. Neeraj Agarwal discusses the ongoing phase 3 CONTACT-02 study of atezolizumab/ cabozantinib in mCRPC - Urology Times

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Sen. Mitt Romney, R-Utah, returns to the chamber from a short break as House impeachment managers present their second day of arguments in the Senate trial of former President Donald Trump, at the Capitol in Washington, Thursday, Feb. 11, 2021. (AP Photo/J. Scott Applewhite)

By Christine B. Helfrich | The Public Forum

| Feb. 13, 2021, 1:00 p.m.

I am sad to see Sen. Mitt Romney joining Sen. Rand Paul in objecting to transgender youth participating in school sports. Romney said, They shouldnt be competing with people who are physiologically in an entirely different category, and I think boys should be competing with boys and girls should be competing with (girls) on the athletic field.

My niece is 62 and athletically gifted. I am 55 and am blessed with double vision. We are physiologically in an entirely different category. My nieces large public school volleyball team took a state championship. My tiny private schools team did not.

Should students be subjected to humiliating physical exams to determine their fitness to participate in team sports?

Sixteen states already allow student trans athletes to compete according to their gender identification in current school records and daily-life activities. The world has not come to an end.

Just because a physician or midwife pronounces a gender at birth, based on a view of external genitalia, doesnt mean that the baby will neatly conform to that designation.

There are many variations in sexual characteristics that include internal genitals, external genitals, gonads, chromosomes, gene expression, hormone levels, and brain structure.

In fact 1.7% of the population meets the definition of intersex, which is about as common as having red hair.

Sen. Romney please get your medical information from someone who doesnt present himself as a board certified ophthalmologist despite his self-created board being out of business since 2011.

Christine B. Helfrich, Millcreek

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Letter: Sad to see Romney objecting to transgender youth participating in sports - Salt Lake Tribune

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Misfolded proteins (orange) in the endoplasmic reticulum may play a role in Wolfram syndromes many symptoms.

By Mitch LeslieFeb. 11, 2021 , 2:00 PM

Maureen Marshall-Doss says the first sign that her vision was deteriorating came when she misidentified the color of a dress. At a backyard get-together about 20 years ago, the Indianapolis resident pointed out an attractive yellow dress another woman was wearing. You see that as yellow? Shes wearing a pink dress, Marshall-Doss recalls her husband responding.

Today, Marshall-Doss is virtually blind. With help from custom made eyeglasses that magnify objects 500 times, I can see shapes, she says. But she can no longer drive and had to quit the job she loved as a school librarian. Along with her dimming vision, she has type 1 diabetes and has lost her sense of taste and smell.

Marshall-Doss is one of 15,000 to 30,000 people around the world with Wolfram syndrome, a genetic disease. For decades, the condition remained enigmatic, untreatable, and fatal. But in the past few years, insights into its mechanism have begun to pay off, leading to the first clinical trials of drugs that might slow the illness and sparking hopes that gene therapy and the CRISPR DNA-editing tool might rectify the underlying genetic flaws. Here is a rare disease that the basic science is telling us how to treat, says physiologist Barbara Ehrlich of the Yale School of Medicine.

The research could also aid more than the relatively few patients with Wolfram syndrome. Driving the diseases many symptoms is a malfunction of the endoplasmic reticulum (ER), the multichambered organelle that serves as a finishing school for many cellular proteins. Known as ER stress, the same problem helps propel far more common illnesses, including type 2 diabetes, amyotrophic lateral sclerosis (ALS), Parkinsons disease, and Alzheimers disease. Wolfram syndrome is the prototype of an endoplasmic reticulum disorder, says medical geneticist Fumihiko Fumi Urano of Washington University School of Medicine in St. Louis. Because Wolfram syndrome is simpler, says Scott Oakes, a cell biologist and pathologist at the University of Chicago, researchers think it could illuminate the mechanisms of other ER-disrupting diseases, which affect hundreds of millions of people worldwide.

In the late 1930s,four children with diabetes were going blind, and doctors were stumped. Like many other people in the United States struggling through the Great Depression, the siblings ate a paltry diet, subsisting on potatoes, bread, oatmeal, and a little milk. But after examining three of the children, Donald Wolfram, a physician at the Mayo Clinic in Rochester, Minnesota, and an ophthalmologist colleague ruled out malnutrition as the cause of their puzzling condition. Lead poisoning and syphilisthough common enoughwerent to blame, either. When Wolfram and his partner wrote up the cases in 1938, they concluded that the symptoms could be manifestations of an hereditary or acquired cerebral lesion.

The physicians were right that the syndrome eventually named for Wolfram is hereditary. Recessive mutations in the gene for a protein called wolframin are responsible for most cases, with glitches in a second gene causing most of the rest. However, the pair was wrong to think the defect lies only in the brain. Instead, the symptoms stem from widespread cell death. Its definitely a disease that affects the whole body, Marshall-Doss says.

The first sign of the illness, appearing when patients are children, is usually diabetes mellitus, or faulty sugar metabolism, sparked by the demise of insulin-secreting beta cells in the pancreas. Most patients also develop the unrelated condition diabetes insipidus, in which the pituitary gland doesnt dole out enough of a hormone that helps control the bodys fluid balance, causing the kidneys to produce huge amounts of urine.

Mutations in the gene for wolframin disrupt the endoplasmic reticulum and lead to cell death throughout the body, causing a range of symptoms.

V. Altounian/Science

Ellie White, 19, of Centennial, Colorado, who was diagnosed with Wolfram syndrome 12 years ago, says she hasnt had a full night of sleep since she was 3 years old. She gets up again and again to use the bathroom and monitor her blood sugar.

Yet she and other patients say that as disruptive as those problems are, they are not the diseases most dismaying consequence. The biggest symptom of Wolfram syndrome that affects me the most is my vision, White says. Because neurons in the optic nerve perish, patients usually go blind within 10 years of their first visual symptoms.

Other neurons die as well. As the disease progresses, brain cells expire, and walking, breathing, and swallowing become difficult. Most people with Wolfram syndrome die before age 40, often because they can no longer breathe. At 57, Marshall-Doss is one of the oldest patients; one of her mutated genes may yield a partly functional version of wolframin, triggering a milder form of the disease, Urano says.

Two advanceshave made it possible to begin to tackle those symptoms. The first was Uranos discovery nearly 20 years ago that linked Wolfram syndrome to ER stress. The ER is where about one-third of a cells newly made proteins fold into the correct shapes and undergo fine-tuning. Cells can develop ER stress whenever they are under duress, such as when they dont have enough oxygen or when misfolded proteins begin to pile up inside the organelle.

In test tube experiments, Urano and his colleagues were measuring the activity of genes to pinpoint which ones help alleviate ER stress. One gene that popped up encodes wolframin, which scientists had shown in 1998 was mutated in patients with Wolfram syndrome. Following up on that finding, Urano and his team determined that wolframin takes part in whats known as the unfolded protein response, which is a mechanism for coping with ER stress in which cells take steps including dialing back protein production.

Scientists think wolframin plays a key role in the unfolded protein response, though they havent nailed down exactly how. When wolframin is impaired, cells become vulnerable to ER stress. And if they cant relieve that stress, they often self-destruct, which could explain why so many neurons and beta cells die in the disease.

Defective wolframin may harm cells in other ways. The ER tends the cells supply of calcium, continually releasing and absorbing the ion to control the amount in the cytoplasm. Changes in calcium levels promote certain cellular activities, including the contraction of heart muscle cells and the release of neurotransmitters by neurons. And wolframin affects calcium regulation.

Beta cells genetically engineered to lack functional wolframin brim with calcium, Ehrlich and colleagues reported in July 2020 in theProceedings of the National Academy of Sciences. When exposed to lots of sugar, the altered cells release less insulin and are more likely to die than healthy beta cells, the team found. The cells share that vulnerability with beta cells from patients with Wolfram syndrome. We think that excess calcium is leading to excess cell death, Ehrlich says.

ER malfunctions could hamstring other organelles as well. The ER donates calcium to the mitochondria, the cells power plants, helping them generate energy. In 2018, a team led by molecular biologist Ccile Delettre and molecular and cellular biologist Benjamin Delprat, both of the French biomedical research agency INSERM, discovered that in cells from patients with Wolfram syndrome, mitochondria receive less calcium from the ER and produce less energy. Those underpowered mitochondria could spur the death of optic nerve cells, the researchers speculate.

Fumihiko Urano holds dantrolene, a muscle relaxant drug he helped test as a treatment for Wolfram syndrome.

The link between ER stress and Wolfram syndrome has been crucial for identifying potential treatments because otherwise we would have nothing to target, Urano says. But a second development was also key, he says: the advocacy and support of patient organizations, such as the Snow Foundation and the Ellie White Foundation, headed by its namesakes mother. The foundations have stepped up with money for lab research and clinical trials when other sources, including government agencies, didnt come through.

Scientists, patients, and their advocates say Urano also deserves much of the credit. Besides treating patients, he heads the international registry of cases and has taken the lead in organizing clinical trials, screening compounds for possible use as treatments, and devising potential therapies. Fumi is clearly the driving force, says Stephanie Snow Gebel, co-founder of the Snow Foundation, who about 10 years ago helped persuade him to forgo a plum job as department chair at a Japanese university and take over the Wolfram program at Washington University.

Patients could soonstart to reap the benefits. In 2016, Urano and colleagues started the worlds first clinical trial for the disease: a phase 1/2 study of dantrolene, an approved muscle relaxant. The molecule was a top performer when they screened 73 potential treatments for their ability to save cells with terminal ER stress. Dantrolene didnt improve vision in the 22 participants, including White, the scientists reported in an October 2020 preprint. But in some patients, beta cells appeared to be working better and releasing more insulin. The drug is safe, but Urano says it will need to be chemically tweaked to target its effects before future trials are warranted.

Researchers are pursuing other possible treatments targeting ER stress or calcium levels. In 2018, U.K. scientists launched a trial that will include 70 patients to evaluate sodium valproate, a therapy for bipolar disorder and epilepsy that, in the lab, prevents cells with faulty wolframin from dying. Last year, another compound that emerged from Uranos screens, the diabetes drug liraglutide, entered a clinical trial. Also last year, an experimental drug developed by Amylyx Pharmaceuticals for Alzheimers disease and ALS received orphan drug designation from the U.S. Food and Drug Administration for Wolfram syndrome because it curbs ER stress. That designation offers tax breaks and other incentives, and it will get trials started sooner, Urano says.

Ehrlich and her team have a candidate of their own that they have begun to test in rodents: the drug ibudilast, which is approved in Japan to treat asthma. The researchers found it reduces calcium levels in beta cells lacking wolframin and boosts their survival and insulin output. New screening projects may reveal still more candidates.

But Urano knows that even if a treatment receives approval, it would be only a Band-Aid for Wolfram syndrome. Hoping to develop a genetic cure, he and colleagues are introducing replacement genes into cells from patients and from mice engineered to replicate the disease. The researchers are endowing the cells with healthy copies of the gene for wolframin or the gene for a protein that reduces ER stress to determine whether they restore cellular function and reduce cell death. At INSERM, Delettre and colleagues are also evaluating whether directing a working gene into optic nerve cells can curtail vision loss in mice with faulty wolframin. The scientists are still gathering data, but early results suggest the treatment can halt the deterioration.

Urano and his collaborators have also turned to the genome editor CRISPR, deploying it to correct the gene defect in patients stem cells and then growing them into beta cells. When the researchers transplanted the revamped cells into mice with diabetes, the animals blood sugar returned to healthy levels, the team reported in April 2020 inScience Translational Medicine.

Stem cell biologist Catherine Verfaillie of KU Leuven is collaborating on the CRISPR research. But she notes that because the faulty wolframin gene affects so many tissues, researchers will have to figure out how to deliver the CRISPR components to most cells in large organs such as the brain and livera prospect she calls pretty daunting. Urano agrees, predicting that CRISPR-based Wolfram therapies might take 10 to 20 years to develop. The alternative approach, gene therapy, could reach clinical trials more quickly, in 3 to 10 years, he says, because researchers have more experience with gene therapy and have created several treatments that have already been approved for other illnesses.

Because it stems from a single genetic glitch, Wolfram syndrome could also help scientists tease out the role of the ER in more complex diseases, including neurological conditions, type 2 diabetes, and cancer. The ER also falters in those diseases, causing cells to die, but the mechanism is harder to discern because they stem from myriad genetic and environmental factors. In Alzheimers disease, for instance, neurons develop ER stress as misfolded proteins accumulate inside and outside the cells.

Besides deepening researchers understanding of other conditions, the research on Wolfram syndrome might even deliver candidate treatments. Everyone would be very excited if we can make advances in targeting ER stress in Wolfram syndrome, Oakes says. It would open up the whole field to doing this in other degenerative diseases.

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The race to treat a rare, fatal syndrome may help others with common disorders like diabetes - Science Magazine

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Buckingham:For starters, I want to emphasize the importance of preventing your skin from aging in general, starting when youre young. When you go outside in the sun, wear protective hats, sunglasses, clothing, and sunscreen.

Your overall health impacts skin health too, so I recommend eating a healthy diet and exercising and not gaining excess weight.

People ask me what the most important components of skincare are, and I think its helpful to view those priorities in the same way we use the food pyramid to eat the right amount of certain foods for a healthy diet.

On that base layer of the pyramid is sunscreen, thats the most important thing. Put that on after using a good facial cleanser to wash away the dirt and oil on your face.

The middle box is a vitamin A derivative such as retinol or Retin-A. We have research that shows if you use these products consistently, you can increase your collagen production and your elastin as well as increasing the epidermal turnover. This can keep the skin looking healthy and less dull. The little box at the top of the pyramid is topical antioxidants like vitamin C, peptides, and pigment-reducing agents.

Once you have that good skincare foundation, there are interventions we can do to reverse the effects of aging.

These include neuromodulators, such as Botox, which can temporarily cause the muscle to not contract as robustly as it should, which reduces the appearance of wrinkles.

Microneedling uses a pen-shaped or roller device with fine needles that penetrate the dermal level and create very tiny punctures in the skin. It heals within a day and promotes the growth of new collagen and elastin. Radiofrequency microneedling uses an actual plastic plate that has needles embedded in it that are stamped or inserted into the skin. While the needles are in the face, it emits radio frequency heat energy, which amplifies the effect of microneedling.

There are several laser treatment options depending on the amount of intervention needed. By using concentrated light in different wavelengths, these treatments can rejuvenate skin by increasing the production of collagen and elastin.

Chemical peels can range from very light peels that cause just a small amount of flaking to more intense peels that can require up to 10 days of recovery and continuous application of healing ointment.

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Ask a Castle Connolly Top Doctor: How Aging and Gravity Affect Your Skin - Everyday Health

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Melatonin is a hormone your body naturally produces to induce sleep. It can also be purchased as an over-the-counter oral supplement to help ease insomnia or reset your body clock after traveling to a different time zone.

Melatonin is generally safe and effective in low doses, but you can overdose which can cause adverse side effects. Here is what you need to know about the side effects of melatonin and how to determine how much to take.

Low doses of melatonin cause relatively few side effects, but it is possible to take too much, says Nicole Avena, PhD, an assistant professor of neuroscience at Mount Sinai School of Medicine in New York City.

Melatonin supplements are not regulated by the US Food and Drug Administration (FDA) and therefore have no standardized dose. The typical dosage found in stores ranges from about one to five mg but can be found in doses as high as 10 mg.

Overdosing on melatonin can lead to side effects, including:

It's hard to say exactly how much melatonin is too much since there is no standardized dose, but it's best to start with the lowest dose possible and work up from there, says Alex Dimitriu, MD, founder of Menlo Park Psychiatry and Sleep Medicine in Menlo Park, California.

There is no known lethal dose of melatonin and no reports of death from taking too much melatonin, Dimitriu says, but taking too much can disrupt your natural circadian rhythm and internal body clock, causing you to actually have more trouble falling asleep.

What the research says: Some studies have shown that cancer patients can benefit from large doses of melatonin ranging from 20mg to 1,000mg, but these doses are generally not recommended for those with no underlying medical conditions.

The effects of taking large doses of melatonin don't appear to be lethal, Dimitriu says, but more research is needed to determine the long-term health implications of melatonin use and the effects of doses larger than 30mg.

The right dose of melatonin for you is the lowest possible dose that can help you sleep with minimal side effects, Dimitriu says. This can vary by individual.

General advice: For most people, a melatonin dose of 0.5 to 5 mg can effectively help them fall asleep. If you find this dose isn't effective, you can increase it from there under the guidance of your doctor.

Melatonin should only be used occasionally unless you have otherwise been instructed by a physician, Avena says. Melatonin is considered safe in low doses for short-term use, but there is little data available on its long-term effects and more research is needed to determine overall safety.

If you find you are relying on melatonin in order to fall asleep every night, Avena recommends lowering your dosage slowly. You may experience a few nights of less than great sleep as your body adjusts.

Relaxation techniques like meditation or breathing exercises can help you fall asleep without the help of a melatonin supplement.

"It is more important to have healthy sleep habits and a regular sleep schedule, exercise, and minimal stress, than to rely on any supplement, including melatonin for sleep," Dimitru says.

Melatonin is not addictive, but taking it every night can cause you to rely on it as part of your bedtime routine.

"People can become psychologically or biologically dependent on any supplement or medication they take, especially with sleep. People start associating substances and behaviors as part of the bedtime routine," Dimitriu says. "The bedtime routine may suffer if some of the parts are missing, whether it be a favorite blanket, bed, or supplement like melatonin."

It is impossible to become physically dependent on melatonin like you could with other substances, like alcohol, Avena says, but you can come to rely on it as a sleep association.

If you find you are needing to take melatonin every night to sleep, you may have another health condition, like anxiety or a sleep disorder that is affecting your ability to sleep, Dimitriu says.

"With melatonin, it is important to maintain healthy habits besides just using a supplement," Dimitriu says, "Our bodies and minds, if healthy and not anxious should be able to sleep naturally."

In small doses, melatonin supplements are a safe and effective way to help you fall asleep, but more research is needed to determine the long-term effects of taking melatonin regularly.

There is no known lethal dosage of melatonin, but taking more than 10 mg can cause side effects, like daytime drowsiness and headaches.

If you are relying on melatonin to sleep, talk with your doctor about ways to improve your sleep hygiene.

More:
Yes, you can overdose on melatonin here's how to find your proper dosage - Insider

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According to cardiologist the prevalence of heart disease has transcended most diseases worldwide to be the leading cause of death. With 17 million people perishing from cardiovascular diseases worldwide, every year, the need to visit a heart doctor is rapidly expanding.

Heart doctors or cardiologists specialize in treating cardiovascular diseases by first observing the heart from multiple levels. An extensive examination is done to determine the root cause of the problem and find ways to heal the patient. Heart doctors also educate their patients on how to best take care of their hearts through lifestyle and diet choices and preventive medicine.

Cardiologists or heart physicians go through medical school before focusing on the heart specialty. With four years of medical school below their belt, they should have an additional three years of medical training specific to cardiology. This training seeks to prepare them for their fellowship, which requires another three years. Overall, a typical cardiologist has the minimum practice of around ten years before they start working professionally. Some have longer due to research degrees or additional fellowships.

Additional skills and interests of heart doctors include the willingness to motivate the patient and make critical judgments about management often after a complex decision making process. Being able to communicate efficiently and effectively with patients and staff is also important. Heart doctors should be confident and always be willing to learn more about the heart and vascular system.

There are telltale symptoms of heart problems that individuals should look out for and aim to seek medical attention as soon as possible:

When experiencing trouble breathing, it is best to see a cardiologist for assessment. Breathing should come naturally, and shortness of breath is a reason for concern.

Low blood flow to the brain can cause loss of consciousness or dizziness and light-headedness. It is important to ensure this is not caused by the heart, ie a heart arrhythmia. A visit to the heart doctor will examine the reasons why the heart isnt pumping blood to the brain.

An unstable sensation, dizziness can indicate the lack of adequate blood flow to the brain, potentially indicating a heart problem.

The occurrence of irregular heartbeats such as palpitations or a racing or erratic heartbeat might indicate heart disorders, warranting the heart physicians visit all the most crucial. Heart flutters also indicate the need for immediate treatment.

This can be due to various reasons, one of which might be angina or heart attack, caused by lack of enough oxygen reaching the heart. It is best to see your physician immediately to correct the diagnosis before the chest pains escalate to something much worse. The pain can sometimes spread to the arm, back, or jaw. Patients can also sometimes experience sweating and shortness of breath alongside the pain.

When visiting the heart physician, the patient will first answer several questions to determine the condition of their heart. These questions range from drug and family history to lifestyle habits, and their list of symptoms.

Next, the physician will conduct a series of tests to try and identify the cardiovascular conditions that the patient might have. These tests include:

An electrocardiogram tests the electrical activity of the heartbeat. This test examines the reason for chestpains, dizziness, and irregular heartbeats. This procedure involves attaching electrodes on your chest, arms and legs, and attaching them to the EKG machine, which records the hearts activity and records the information on a graph. This procedure is painless and takes around ten to fifteen minutes.

This test seeks to understand how the heart performs at a time when it requires the most blood flow, stress. The patient is attached to an EKG machine while on a treadmill. The heart physician monitors the ECG or echocardiogram to determine whether enough oxygen through proper blood flow is directed towards the heart.

The patient lies on a bed and a technician or heart specialist uses an ultrasound machine to look at the structure and function of the heart, and its valves. The test takes 20-30 minutes and no X-rays are used.

This procedure involves introducing a catheter to the blood vessel until it reaches the heart to diagnose heart problems. The catheter is injected with a dye, which exposes irregularities or blockages of the heart arteries.

After these tests, the heart physician will diagnose the results and advise on a suitable treatment plan. Depending on the heart condition, the heart physician will prescribe medications and advice on preventive and lifestyle changes. More severe complications will require heart surgery, depending on the situation.

It is paramount to maintain healthy lifestyle practices that will help prevent a visit to the cardiologist. Such practices include:

Read more:
All You Need to Know About a Heart Doctor Before Your Visit - Chiang Rai Times

Recommendation and review posted by Bethany Smith

Happy national "Satisfied With Being Single Day"!

I love that this falls right before Valentine's Day. It's a great way to embrace the solo life. It's also a great day to print out a picture of your ex. I know it sounds counter productive, but it's actually going to benefit you this weekend.

The benefit? Free wings. What's better than that? Hooters does this hilarious thing every year where they want to help you get over your ex, and in return they'll also give you some free food.

So I went to go check if the Hooters in Rockford is one of the participating locations, and it led me to a virtual survey about my ex. So of course I took it. Here's what the survey looked like -

So after I chose some of the hilarious options, they lead me here.

Then they made me pick a picture, and I wasn't really trying to shred an ex. So I picked a picture of the 97ZOK logo (oops).

Well, here we go.

I'M SORRY 97ZOK! I didn't mean it!

After all that virtual shredding, I was finally prompted to enter my information for a coupon to the Rockford location. So if you have an ex you're ready to get over, and you're also a wing lover, then this is the perfect way to celebrate Valentine's Day.

Also, I still recommend printing out a picture to ACTUALLY shred. Sounds like a great way to get over someone.

H/T DELISH

See the article here:
Hooters Will Give You Free Wings For Shredding a Pic of Your Ex - q985online.com

Recommendation and review posted by Bethany Smith

Though a peanut allergy is generally considered a pediatric issue, many people don't develop one until after turning 18 years old.

More than 800,000 of the 4.6 million U.S. adults who have peanut allergies didn't develop them until after reaching adulthood, according to a new study. That equates to 17% a larger percentage than previously believed.

The reasons behind late-onset peanut allergies are not completely understood, but the Northwestern University researchers who conducted the study suggest environmental factors and hormone fluctuations may play a role.

Other experts said a delayed or extended period of sensitization to peanuts could also be to blame or even a cross-reaction to some other allergens such as pollen. Research is ongoing to improve diagnosis, management and treatment of peanut allergies.

"Given the high prevalence of peanut allergy among U.S. adults, additional therapies are needed to help address this growing burden of disease,"Dr. Ruchi Gupta, director of the Center for Food Allergy and Asthma Research at Northwestern University,toldUPI.

The study's findings, published inThe Journal of Allergy and Clinical Immunology, stem from a survey of more than 40,000 adults.

Though 2.9% of respondents reported having peanut allergies, researchers found only 1.8% actually had convincing peanut allergies.Gupta said it is important for adults who think they have developed peanut allergies to be diagnosed by a physician instead of just avoiding peanuts.

Their doctors can teach them how best to avoid exposure and prescribe epinephrine, an emergency treatment for anaphylaxis, a severe allergic reaction.

Gupta also toldCNNthat more adults than children have peanut allergies. Only 15 to 20% of children with a peanut allergy will outgrow it by adulthood.

People who said they developed peanut allergies in adulthood were less likely to have been diagnosed by a physician compared to adults whose allergies emerged during childhood, researchers found.

They also were more likely to report multiple food allergies.They were less likely to have an epinephrine prescription 44% compared to 56%.

A food allergy develops when the immune system mistakenly labels the food as a threat and launches an attack against it, Harvard Health experts say.

For people with peanut allergies, even a small amount of peanuts can cause a serious, possibly life-threatening reaction. Symptoms usually start minutes after exposure, according to the Mayo Clinic.

The most common symptoms include skin reactions such as hives or redness, an itching or tingling in the mouth or throat, nausea or vomiting, tightening of the throat and wheezing.

Any severe reaction requires treatment with an epinephrine autoinjector and an emergency department visit. Anaphylaxis symptoms include trouble breathing, a sharp drop in blood pressure, a rapid pulse, dizziness and loss of consciousness.

Peanut allergies are a leading cause of fatal and near-fatal anaphylaxis. People who suspect they may have peanut allergies should get evaluated by their physicians as soon as possible.

The U.S. Food and Drug Administration has not approved any therapies for adults with peanut allergies, An oral immunotherapy has been approved for patients 4 to 17 years of age to help desensitize them to peanuts.

See more here:
More than 4.6 million US adults have peanut allergies, Northwestern University study finds - PhillyVoice.com

Recommendation and review posted by Bethany Smith