Clinical Trial Alert: Phase 1/2 Study of AT845 in Late-Onset Pompe Disease

Researchers at Audentes Therapeutics, an Astellas company, are seeking individuals living with late-onset Pompe disease (LOPD) to participate in a phase 1/2 study to confirm safety and efficacy of the investigational drug AT845. This gene-replacement therapy may offer the benefit of long-term improvement of motor and respiratory function and quality of life in adults living with . . .

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Clinical Trial Alert: Observational Study to Identify Biomarkers in Individuals Receiving Radicava for ALS

Researchers at Mitsubishi Tanabe Pharmaceutical America (MTPA) are seeking individuals living with amyotrophic lateral sclerosis (ALS) to participate in a six-month observational study. This study is designed to identify biomarkers to serve as quantifiable, biological, non-clinical measures of Radicava (edaravone) effects on ALS. Observational measures might include assessment of vitals; blood and urine collection; safety assessments; use . . .

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Clinical Trial Alert: Phase 3 Study of Zilucoplan in Individuals With Generalized Myasthenia Gravis

Researchers at Ra Pharmaceuticals (now part of UCB BioSciences) are seeking individuals living with generalized myasthenia gravis (gMG) to participate in a phase 3 study to confirm safety, tolerability, and efficacy of the investigational drug Zilucoplan. This therapy may improve muscle function by blocking the autoimmune reaction that destroys the junction between nerves and muscles in . . .

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FDA Approves Sarepta Therapeutics’ Amondys 45 for Treatment of DMD Amenable to Skipping Exon 45

On Feb. 25, the US Food and Drug Administration (FDA) granted accelerated approval to casimersen (Amondys 45) for the treatment of Duchenne muscular dystrophy (DMD) in patients amenable to skipping exon 45. It is the fourth exon-skipping, disease-modifying drug to treat DMD, the most common childhood form of muscular dystrophy. The drug is administered by intravenous . . .

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Clinical Trial Alert: Phase 3 Study of Rozanolixizumab in Individuals With Generalized Myasthenia Gravis

Researchers at UCB BioSciences are seeking individuals living with generalized myasthenia gravis (gMG) to participate in a phase 3 study. The objective of the study is to confirm the clinical efficacy and to assess safety and tolerability of rozanolixizumab. The therapy may help reduce the typical signs and symptoms of gMG, which are caused by autoantibodies . . .

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MDA Welcomes Dr. Don Wood as its New President and CEO

Steve Jobs said, “The only way to do great work is to love what you do.” And MDA’s new president and CEO, Donald S. Wood, PhD, surely loves what he does. “The mission of this organization has been a lifelong passion for me,” Dr. Wood says. After receiving his doctorate in Physiology from Washington State . . .

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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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