The mother of a four-year-old boy with a rare muscle disease says screening newborn babies for the condition could "save ...

"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.

Approval is supported by data from the DEVOTE study which showed the benefit of the SPINRAZA 50 mg and 28 mg regimen in both treatment-naïve and ...

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 ...

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 ...

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 ...