"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.

Please provide your email address to receive an email when new articles are posted on . At 5 years, 91% of pediatric patients treated with Evrysdi were alive, 81% without permanent ventilation. Data ...

We were unable to process your request. Please try again later. If you continue to have this issue please contact customerservice@slackinc.com. Patients treated with onasemnogene abeparvovec recorded ...

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

Spinal muscular atrophy is a debilitating genetic condition that’s usually fatal by a few years of age. But an intriguing case study might demonstrate a simple new treatment, with a child showing no ...

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 ...