Novartis Secures FDA Approval of ITVISMA for Children Two Years and Older, Teens, and Adults with Spinal Muscular Atrophy (SMA)
Novartis has announced a significant expansion in the treatment landscape for Spinal Muscular Atrophy (SMA) with the FDA approval of ITVISMA (onasemnogene abeparvovec-brve). This one-time gene replacement therapy is now approved for children two years and older, teens, and adults with a confirmed mutation in the Survival Motor Neuron 1 (SMN1) gene.
SMA is a devastating, rare, genetic neuromuscular disorder caused by a missing or mutated SMN1 gene, leading to deficient SMN protein production, progressive motor neuron loss, muscle weaknes...