Simply Stated: What is Myasthenia Gravis (MG)?

Myasthenia gravis (MG) is a chronic neuromuscular disease characterized by muscle weakness that worsens after activity and improves after rest. MG is caused by an autoimmune reaction in which the body’s immune system attacks its own tissues, interrupting the connection between nerves and muscles (the neuromuscular junction). MG can occur at any age and affects . . .

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Eledon Announces Positive Topline Results from a Phase 2a Study of Tegoprubart to Treat ALS

On May 31, Eledon announced positive topline results from its Phase 2a clinical trial of the investigational therapy tegoprubart (formerly AT-1501) for treatment of amyotrophic lateral sclerosis (ALS). The primary endpoints of the study, assessment of safety and tolerability, demonstrated an encouraging safety profile that supports further investigation. Secondary endpoints showed that the drug engaged . . .

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Simply Stated: What is Friedreich’s Ataxia (FRDA)?

Friedreich’s ataxia (FRDA) is an inherited neuromuscular disease that primarily impacts the nervous system and heart and affects about one in 50,000 people worldwide. FRDA typically appears in people before the age of 25 years and is characterized by a slow, progressive loss of limb coordination (ataxia) and effects on speech and swallowing. While there . . .

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Clinical Trial Alert: Phase 1b/2a Study of WVE-N531 in a Subset of People Living with DMD

Researchers at Wave Life Sciences are seeking boys living with DMD caused by gene mutations amenable to exon 53 skipping interventions to participate in a phase 1b/2a clinical trial. This trial will evaluate the safety, effectiveness, and correct dosage of the investigational drug WVE-N531. WVE-N531 is an exon-skipping drug designed to promote skipping over a section . . .

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Clinical Trial Alert: Phase 3b Study of Oral Edaravone in Adults with ALS

Researchers at Mitsubishi Tanabe Pharma Development America, Inc. (MTPA) are seeking adults living with amyotrophic lateral sclerosis (ALS) to participate in a phase 3b clinical trial to evaluate the safety and efficacy of oral edaravone to treat ALS. Edaravone (brand name Radicava) was designed to help protect cells from damage caused by free radicals, and was . . .

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Capricor Announces Positive Results from its Phase 2 Study of CAP-1002 to Treat DMD

On September 24, Capricor Therapeutics announced positive results at the World Muscle Society Virtual Congress from its phase 2 HOPE-2 trial of the investigational therapy CAP-1002 for treatment of people in advanced stages of Duchenne muscular dystrophy (DMD). The primary and secondary endpoints of the study, the improvement of upper limb and cardiac function, were met, . . .

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How Expedited Drug Approval Impacts the Neuromuscular Disease Treatment Landscape

The urgency of the COVID-19 pandemic has transformed the drug-development landscape, driving rapid development, US review, and, in one case, new therapeutic approval. To bring critical drugs to market, the US Food and Drug Administration (FDA) is utilizing a special emergency program announced in April 2020 to accelerate approval of promising coronavirus therapies, an initiative . . .

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MDA’s 2021 Advocacy Plan

2021 is a new year: The country has a new administration, and a new Congress has been sworn in. What isn’t new is MDA’s commitment to working tirelessly in Washington, DC, to transform the lives of people living with neuromuscular diseases (NMDs). Here’s a sneak peek at some of the issues we’ll be working on . . .

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MDA Advocacy in These Uncertain Times

We know families across the country, especially those in the neuromuscular disease (NMD) community, are concerned about the spread of coronavirus and its associated disease, COVID-19, and the impact on their loved ones. In these uncertain times, MDA’s advocacy team will not stop serving and amplifying the voices of those in the NMD community, especially . . .

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