![FDA, FTC and DOJ Enforcement of Medical Device Regulations FDA, FTC and DOJ Enforcement of Medical Device Regulations](https://www.fdanews.com/ext/resources/Book-Covers-2/BFFDEMDR-COVER.png?height=200&t=1685733565&width=200)
Home » FDA Approves Gene Therapy for Pediatric Spinal Muscular Atrophy
FDA Approves Gene Therapy for Pediatric Spinal Muscular Atrophy
![Purple_Approved_Stamp.gif](https://www.fdanews.com/ext/resources/test/Drug-Images4/Purple_Approved_Stamp.gif?t=1576639773&width=430)
May 31, 2019
The FDA approved AveXis’ Zolgensma (onasemnogene abeparvovec-xioi) as the first gene therapy approved to treat children less than two years of age with spinal muscular atrophy (SMA), a leading genetic cause of infant mortality.
Zolgensma is an adeno-associated virus vector-based gene therapy that targets the cause of SMA, a rare genetic disease caused by a mutation in the survival motor neuron 1 (SMN1) gene. Children with the condition have problems holding their head up, swallowing and breathing.
“Today’s approval marks another milestone in the transformational power of gene and cell therapies to treat a wide range of diseases,” said Acting FDA Commissioner Ned Sharpless.
Upcoming Events
-
21Oct