The first patient has been dosed in a Phase 1/2 study of GB221, a next-generation gene therapy for SMA type 1.

Novartis has received approval from the U.S. Food and Drug Administration for Itvisma, a treatment for spinal muscular atrophy. Dr. John Day is professor of neurology and pediatrics, director of the ...

PHILADELPHIA--(BUSINESS WIRE)--Exegenesis Bio, a rapidly growing global genetic medicines company, is pleased to announce the presentation of clinical efficacy and safety data from its EXG001-307 ...

A positive newborn screening for spinal muscular atrophy (SMA) is currently considered a medical emergency. Without early treatment, severe disability or death in infancy are likely. However, research ...

This story was originally published on BioPharma Dive. To receive daily news and insights, subscribe to our free daily BioPharma Dive newsletter. The Food and Drug Administration on Monday approved a ...

Findings from a new study in the Journal of Neuromuscular Diseases, published by IOS Press, demonstrate the effectiveness of disease-modifying treatments (DMTs) in infants with spinal muscular atrophy ...