Muscular Dystrophy Association
Recent News About Muscular Dystrophy Association
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MUSCULAR DYSTROPHY ASSOCIATION: What You Need to Know About Open Enrollment in 2020
Open enrollment — the period each year when people are eligible to purchase new insurance or make changes to their existing insurance plans — is upon us.
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MUSCULAR DYSTROPHY ASSOCIATION: Everyone Invited to Participate in Survey About Improving Genetic Counseling
In order to make progress toward ensuring all future genetic counselors possess the necessary skills to improve the quality of services provided to families and individuals, researchers at the University of Texas Health Sciences Center in Houston are seeking participants for an online survey.
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MUSCULAR DYSTROPHY ASSOCIATION: FDA Approves Genentech’s Evrysdi for Treatment of SMA in Pediatric and Adult Patients
On Aug. 7, the US Food and Drug Administration (FDA) granted approval of risdiplam (Evrysdi) for the treatment of spinal muscular atrophy (SMA) in pediatric and adult patients. It is the third disease-modifying therapy approved to treat SMA, the leading genetic cause of infant death.
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MUSCULAR DYSTROPHY ASSOCIATION: A Q&A With MDA Let’s Play Community Manager Paul Scherer
In the gaming world, Paul Scherer goes by Puzzle. And as community manager of MDA Let’s Play, our dedicated community gaming and fundraising platform, he anticipates technical issues and seeks solutions to player requests to put together an experience that, for MDA gamers, is as seamless as it is satisfying.
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MUSCULAR DYSTROPHY ASSOCIATION: 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.
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MUSCULAR DYSTROPHY ASSOCIATION: Sarepta Therapeutics Announces Positive Results from the SRP-9003 Gene Therapy Trial to Treat LGMD2E
Sarepta Therapeutics Announces Positive Results from the SRP-9003 Gene Therapy Trial to Treat LGMD2E
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MUSCULAR DYSTROPHY ASSOCIATION: NS Pharma Announces Positive Results from Phase 2 Study of Viltolarsen in DMD
NS Pharma Announces Positive Results from Phase 2 Study of Viltolarsen in DMD
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MUSCULAR DYSTROPHY ASSOCIATION: Acceleron Pharma Announces Discontinuation of Clinical Trials Program for CMT
On March 9, Acceleron Pharma, Inc., announced that a phase 2 clinical trial of its therapy under development, ACE-083, for the treatment of Charcot-Marie-Tooth disease (CMT) did not show statistically significant improvements in functional tests compared to placebo.
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MUSCULAR DYSTROPHY ASSOCIATION: Clinical Trial Alert: LGMD2I Participants Sought for a Phase 3 Study
Researchers at PTC Therapeutics are looking for individuals with limb-girdle muscular dystrophy type 2I (LGMD2I) to participate in a phase 3 study.