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. Buying health insurance can be confusing, but it’s critically important that everyone in the neuromuscular community have coverage. We are pleased to share some FAQs below to help . . .

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Scholar Rock Announces Positive Results from Interim Analysis of TOPAZ Phase 2 Study of SRK-015 to Treat SMA

On Oct. 27, Scholar Rock announced positive results from an interim analysis of its phase 2 clinical trial evaluating SRK-015, a therapy for treating spinal muscular atrophy (SMA). After six months of treatment with SRK-015, patients with SMA types 2 and 3 demonstrated significant motor function improvements, as measured by the Hammersmith scale, with higher dose . . .

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Amylyx Announces More Results from Study of AMX0035 to Treat ALS

On Oct. 16, Amylyx announced an update from its CENTAUR trial evaluating the effects of the investigational drug AMX0035 (a combination of the small molecules sodium phenylbutyrate and taurursodiol) on overall survival in people with amyotrophic lateral sclerosis (ALS). The current study followed each participant in the CENTAUR trial for up to three years from . . .

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FDA Accepts Sarepta Therapeutics’ New Drug Application for DMD Treatment Casimersen

On Aug. 25, Sarepta Therapeutics announced that the US Food and Drug Administration (FDA) accepted the company’s New Drug Application (NDA) seeking accelerated approval for casimersen (SRP-4045), an investigational therapy for Duchenne muscular dystrophy (DMD) amenable to exon 45 skipping. The FDA has granted Sarepta’s application Priority Review Status, meaning that a decision is expected . . .

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FDA Approves NS Pharma’s Viltepso for Treatment of DMD Amenable to Exon 53 Skipping

Today, the US Food and Drug Administration (FDA) granted accelerated approval to viltolarsen (Viltepso) for the treatment of Duchenne muscular dystrophy (DMD) in patients amenable to skipping exon 53. It is the third exon-skipping, disease-modifying drug to treat DMD, the most common childhood form of muscular dystrophy. The drug is administered by intravenous infusion. Viltepso will . . .

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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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