July 30, 2019
The U.S. Food and Drug Administration approved Zolgensma (onasemnogene abeparvovec-xioi), the first gene therapy approved to treat children less than two years of age with spinal muscular atrophy (SMA), the most severe form of SMA and a leading genetic cause of infant mortality.
Related Diseases:
Spinal muscular atrophy
Spinal muscular atrophy 1
Spinal muscular atrophy type 2