Media Focus

Jun 11, 2019

FDA Approves Zolgensma, first gene therapy for SMA

Zolgensma, a first gene therapy for spinal muscular atrophy - and first for any chronic neurologic disease - is now an approved and potential "one-time" intravenous treatment for pre-symptomatic newborns through 2-year-olds with any type of SMA, the U.S. Food and Drug Administration (FDA) announced today, issuing an historic decision.