About SMA:
SMA Press
About SMA: SMA Press
Scholar Rock announced that ISEMBYLD™ (apitegromab-mstn) was approved by the U.S. Food and Drug Administration (FDA) for the treatment of Spinal Muscular Atrophy (SMA) in adults and children 2 years of age and older who are currently receiving an SMN2-targeted treatment. ISEMBYLD showed a clinically meaningful improvement in motor function in the Phase 3 SAPPHIRE…
The SMA Foundation’s partners, Roche and PTC Therapeutics, announced today that the FDA has approved a label extension for Evrysdi(risdiplam) to include babies under two months old with SMA. The approval is based on interim efficacy and safety data from the RAINBOWFISH study in newborns, which showed that pre-symptomatic babies treated with Evrysdi for at least one…
Columbia University Press: “Columbia University & Columbia-Presbyterian Med Ctr Open New SMA Clinic”
WHEN & WHERE: Tuesday, September 17, Noon Harkness Pavilion 180 Fort Washington Avenue New York NY WHAT: The Departments of Neurology Columbia University and at Columbia Presbyterian Medical Center are celebrating the opening of the SMA (spinal muscular atrophy) Clinic, a center devoted to the treatment and management of a group of diseases that affect…