"Our preliminary findings show that German children with SMA, despite significant physical disability, have surprisingly good subjective HRQoL." – Erik Landfeldt, MSc, PhD Previous studies have found ...

The least-squares mean difference in the Hammersmith Functional Motor Scale-Expanded change from baseline at 12 months was 1.8 points for those 2 to 12 years receiving apitegromab vs placebo.

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 ...

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 ...

We were unable to process your request. Please try again later. If you continue to have this issue please contact [email protected]. Topline results from a phase 3 clinical trial showed an ...

Spinal muscular atrophy with respiratory distress (SMARD) is a rare genetic condition that typically affects infants and children. It causes muscle weakness and breathing problems. Spinal muscular ...

Scholar Rock Holding Corp.’s stock soared more than 315% Monday to put it on track for its biggest ever one-day gain, after the clinical-stage biotech company said a late-stage trial of a treatment ...