First Gene Therapy for Spinal Muscular Atrophy

AveXis, a Novartis company,receives FDA approval for Zolgensma®, the first and only gene therapy for pediatric patients with spinal muscular atrophy (SMA). Zolgensma is approved for the treatment of children less than 2 years of age with spinal muscular atrophy (SMA). Zolgensma® replaces the defective or missing SMN1 gene to halt disease progression with a single, one-time infusion.