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...
SMA Care Has Significant Financial, Career, Social, and Psychological Impact on Families, Study Reports

SMA Care Has Significant Financial, Career, Social, and Psychological Impact on Families, Study Reports

Taking care of a child with spinal muscular atrophy (SMA) significantly affects several aspects of  families’ lives, including finances, career choices, social life, and mental health, according to an Australian study. The study, “Financial, opportunity and...