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Researchers at PTC Therapeutics are looking for individuals with limb-girdle muscular dystrophy type 2I (LGMD2I) to participate in a phase 3 study. The goal of the study is to evaluate…

Researchers at Mitsubishi Tanabe Pharma Development America are looking for participants with amyotrophic lateral sclerosis (ALS) to participate in a phase 3 study to evaluate the safety of oral edaravone…

On Jan. 22, Genentech, a member of the Roche Group, announced positive results from the second part of its pivotal phase 2/3 FIREFISH clinical trial assessing risdiplam in infants age…

Researchers at Sarepta Pharmaceuticals are looking for pediatric patients with limb-girdle muscular dystrophy type 2E (LGMD2E; aka beta-sarcoglycan deficiency) to participate in a phase 1/2a study. The goal of the…

In 1950, when the Muscular Dystrophy Association was founded, muscular dystrophy and neuromuscular disease weren’t well known or well understood. But they were, as today, affecting an entire community of…

Researchers at Scholar Rock are looking for individuals with spinal muscular atrophy (SMA) type 3 to participate in a phase 2 study. The goal of the study is to evaluate…

It might only be a month into 2020, but MDA’s advocacy team and grassroots advocates are already making their voices heard. We expect the next 12 months to be crucial…

Australian company Antisense Therapeutics announced encouraging results from its phase 2, open-label clinical trial of the company’s investigational therapy ATL1102 for treating Duchenne muscular dystrophy (DMD). The study met its…

This public health program makes early treatment possible for babies born with neuromuscular diseases

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