"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 ...

A Prescription Drug User Fee Act target date of September 22, 2025 has been set for the application. The Food and Drug Administration (FDA) has accepted for Priority Review the Biologics License ...

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.

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

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 ...

"Our study demonstrated that profound impairments in swallowing are extremely common within the first year of life. This of course has tremendous clinical relevance as it highlights the importance of ...

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 ...

Elaine Chen covers biotech, co-writes The Readout newsletter, and co-hosts STAT’s weekly biotech podcast, The Readout Loud. You can reach Elaine on Signal at elaineywchen.70. The biotech company ...