FDA Approves AveXis’ Gene Therapy Zolgensma to Treat SMA in Children Up to Age 2

FDA Approves AveXis’ Gene Therapy Zolgensma to Treat SMA in Children Up to Age 2

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...