"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 ...

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 ...

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 ...

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.

Jason Mast is a general assignment reporter at STAT focused on the science behind new medicines and the systems and people that decide whether that science ever reaches patients. You can reach Jason ...

Oral risdiplam (Evrysdi, Genentech) started in the first 6 weeks of life let most infants with presymptomatic spinal muscular atrophy (SMA) reach motor milestones typical of healthy babies, results of ...

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 ...