Emma Ciafaloni, MD, FAAN, explores the groundbreaking approval of onasemnogene abeparvovec-xioi for spinal muscular atrophy and its impact on the treatment paradigm for this rare disease. This is a ...

SCHAUMBURG, Ill., Nov. 26, 2025 /PRNewswire/ -- Cure SMA, the leading nonprofit organization dedicated to supporting those impacted by spinal muscular atrophy (SMA), welcomes the FDA approval of ...

Forbes contributors publish independent expert analyses and insights. Spinal muscular atrophy affects the nerves that control muscle movement, leading to progressive weakening. As a result, infants ...

Biogen recorded declining sales for Spinraza in each of the four quarters of 2025, but the Massachusetts drugmaker sees ...

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 ...

The first prenatal treatment for spinal muscular atrophy showed promise in a single case report. Risdiplam was given to the mother during pregnancy and to the child after birth. More than 2 years ...

PORTLAND, Ore. (KOIN) – August is Spinal Muscular Atrophy awareness month, and the disease’s first ever at-home treatment is having a major impact. SMA is a rare nerve disorder that affects motor ...

Scholar Rock's biologics license application for apitegromab was accepted by the Food and Drug Administration. The late-stage biopharmaceutical company said the regulator will review the application ...

The MarketWatch News Department was not involved in the creation of this content. SCHAUMBURG, Ill., Nov. 26, 2025 /PRNewswire/ -- Cure SMA, the leading nonprofit organization dedicated to supporting ...