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Category Archives: Muscular Dystrophy Treatment

Dyne Therapeutics Announces Appointment of Molly White as Vice President, Medical Communications and Advocacy – Yahoo Finance

Posted: January 13, 2020 at 6:47 pm

Former chief executive officer of Myotonic brings extensive experience in patient advocacy and community engagement Dyne Therapeutics, a biotechnology company pioneering targeted therapies for patients with serious muscle diseases, today announced the appointment of Molly White as Vice President, Medical Communications and Advocacy. Continue reading

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Can this San Diego startup mimic exercise and fasting with a pill? Epirium raises $85M to find out – The San Diego Union-Tribune

Posted: at 6:47 pm

A biotech startup in La Jolla has raised $85 million from notable science investors to investigate its idea for treating age-related diseases: a pill that mimics the effects of exercise and fasting. The company, called Epirium Bio, says its scientists have discovered a new human hormone, which, when influenced, can trigger the production of more mitochondria Continue reading

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Sarepta Therapeutics Appoints Renowned Biotech Executive John C. Martin to its Board of Directors – Yahoo Finance

Posted: at 6:47 pm

CAMBRIDGE, Mass., Jan. 13, 2020 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc. (SRPT), the leader in precision genetic medicine for rare diseases, announced that it has appointed John C Continue reading

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Inside Montel Williams’ journey from long-undiagnosed patient to MS activist – Media News – MM&M – Medical Marketing and Media

Posted: at 6:47 pm

Given his level of education, the resources at his disposal and his media celebrity, one would place Montel Williams near the bottom of any list of individuals likely to endure a missed diagnosis for two full decades. And yet there he was during the bulk of the 1980s and 90s, enduring pain in his lower extremities and temporary blindness in one eye as doctor after doctor failed to diagnose him with relapsing-remitting multiple sclerosis. That he lived like this while, among other things, serving in the Navy, hosting a syndicated TV talk show and becoming a father to four children, ranks as a fairly amazing achievement in retrospect. Continue reading

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Innovation in New Drug Approvals of 2019 Advances Patient Care – FDA.gov

Posted: at 6:47 pm

By: Janet Woodcock, M.D., Director, Center for Drug Evaluation and Research Happy New Year! The beginning of a new year is always a rewarding time for the many dedicated staff at the FDA's Center for Drug Evaluation and Research (CDER) who work on approving new drug therapies. We look back over the past year and see how the work we did came together to help patients in need one of the main reasons why many of us, including me, have chosen a career in regulatory science and public health. Continue reading

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Marketers: Focus on fundamentals to make a return this year – News – MM&M – Medical Marketing and Media

Posted: at 6:47 pm

News stories from a certain Las Vegas electronics confab this week promise us all a future of health-oriented wearables and cyber companions. Among this years crop of gadgetry is a neuro-feedback headset designed to combat insomnia and a wristband that scans food and tells you what to eat based on your DNA. Make no mistake Continue reading

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Ask the Pharmacist: Medications introduced in 2019 – Marco News

Posted: January 4, 2020 at 9:41 pm

Suzy Cohen, Columnist Published 5:02 a.m. ET Dec. 30, 2019 Dear Readers, its time for my annual article regarding new medications of the year. Continue reading

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Roche and Sarepta Partner on Duchenne Muscular Dystrophy Therapy – BioSpace

Posted: December 28, 2019 at 7:42 am

Sarepta Therapeutics and Roche inked a licensing deal that gives Roche exclusive commercial rights to SRP-9001, Sareptas investigation gene therapy for Duchenne muscular dystrophy (DMD), outside the U.S. DMD is a muscle wasting disease caused by mutations in the dystrophin gene. Continue reading

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New HealthWell Foundation Fund Offers Financial Assistance to People Living with Lambert-Eaton Myasthenic Syndrome (LEMS) – BioSpace

Posted: at 7:42 am

GERMANTOWN, Md., Dec. 27, 2019 /PRNewswire/ -- The HealthWell Foundation, an independent non-profit that provides a financial lifeline for inadequately insured Americans, has opened a new fund to provide copayment and premium assistance to people living with Lambert-Eaton Myasthenic Syndrome (LEMS). Continue reading

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Comedy show, auction to benefit Doylestown natives battle with ALS – The Intelligencer

Posted: at 7:42 am

Jay Smith, a Central Bucks West graduate now living in Texas, continues his fight against ALS, with which he was diagnosed in 2014. Continue reading

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