Panelists discuss how the current spinal muscular atrophy (SMA) treatment landscape includes 3 options: gene therapy (onasemnogene abeparvovec [Zolgensma]) for younger patients and 2 splice modifiers ...

University of Pittsburgh School of Medicine researchers carrying out a small pilot clinical trial demonstrating that a drug-free, minimally invasive intervention targets the root cause of progressive ...

Spinal muscular atrophy is a genetically inherited disorder that causes muscle weakness. Adults can get spinal muscular atrophy, but it’s rare in adults and progresses slowly. It doesn’t typically ...

Spinal muscular atrophy (SMA) is a genetic neuromuscular disease affecting specialized nerve cells that control voluntary muscle movement, according to the Muscular Dystrophy Association (MDA). It can ...

Forbes contributors publish independent expert analyses and insights. Spinal muscular atrophy affects the nerves that control muscle movement, leading to progressive weakening. As a result, infants ...

Panelists discuss how spinal muscular atrophy is an autosomal recessive genetic disease affecting motor neurons with 3 currently approved disease-modifying therapies that restore SMN protein ...