Biogen Announces Positive Results from Phase 1/2 Study of Tofersen to Treat a Genetic Form of ALS

On July 8, Biogen announced positive results from its phase 1/2 clinical trial of the investigational therapy tofersen (BIIB067) for treatment of amyotrophic lateral sclerosis (ALS) caused by mutation of the superoxide dismutase 1 gene (SOD1). The primary endpoints of the study — assessment of safety, tolerability, and drug dynamics in the body — demonstrated . . .

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How MDA is Supporting LGMD Research and Care in 2020

On July 18, MDA is hosting a free virtual Engage Limb-Girdle Muscular Dystrophy Symposium, a half-day educational event featuring key experts who will speak on topics including the latest in genetics and research in limb-girdle muscular dystrophy (LGMD). MDA has a longstanding commitment to finding treatments for the more than 30 subtypes of LGMD; since . . .

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Sarepta Therapeutics Announces Positive Results from the SRP-9001 Gene Therapy Trial to Treat DMD

On June 15, Sarepta Therapeutics announced positive results from its phase 1/2a study of SRP-9001 gene therapy to treat Duchenne muscular dystrophy (DMD). Data from four patients indicated that a single intravenous infusion of SRP-9001 was safe and well tolerated, with no serious adverse events. Additionally, all participants demonstrated improvements across multiple efficacy-related endpoints, including . . .

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Momenta Pharmaceuticals Announces Positive Results from Phase 2 Vivacity-MG Study of Nipocalimab for Treatment of Generalized Myasthenia Gravis

On June 15, Momenta Pharmaceuticals announced positive results from its phase 2 Vivacity-MG trial assessing treatment with the company’s investigational therapeutic nipocalimab (M281) in patients with generalized myasthenia gravis (gMG). Preliminary data indicated that treatment with nipocalimab, using four different dosing protocols over an eight-week period, resulted in improvements in patients’ Myasthenia Gravis Activities of . . .

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Five Questions With CMT Researcher Stephan Züchner

Stephan Züchner, MD, PhD, professor for Human Genetics and Neurology, Chair of the Human Genetics Department, and codirector of the John P. Hussman Institute for Human Genomics at the University of Miami Miller School of Medicine, recently discovered mutations in the sorbitol dehydrogenase gene (SORD) that cause a recessive, axonal form of Charcot-Marie-Tooth disease (CMT) . . .

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Sarepta Therapeutics Announces Positive Results from the SRP-9003 Gene Therapy Trial to Treat LGMD2E

On June 8, Sarepta Therapeutics announced positive results from its first study in human patients of SRP-9003, a gene therapy designed to treat limb-girdle muscular dystrophy (LGMD) type 2E. Data from low-dose and high-dose patient cohorts indicated that a single intravenous infusion of SRP-9003 was well tolerated, with only one serious adverse event occurring in . . .

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ReveraGen BioPharma Announces Completion of Vamorolone Study in 41 DMD Patients

On June 2, ReveraGen BioPharma announced the completion of two-and-a-half years of vamorolone treatment in 41 boys with Duchenne muscular dystrophy (DMD). The long-term data from this study indicated that daily oral administration of vamorolone, even at its highest tested dose, was safe and well tolerated with no serious adverse events. Preliminary data have shown . . .

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A Letter from Case Worker Jana Unislawski to the MDA Community: How to Create an Emergency Care Plan for the COVID-19 Era

MDA Care Center providers across the country are working on the frontlines of the novel coronavirus pandemic. Every day, they’re fielding patient questions and finding new ways to practice medicine in a new time. Today, Jana Unislawski, a clinical case worker and coordinator of the Memory Disorder Clinic at University of Florida Health, shares how . . .

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