SMA in Mid-March: Clinical Practice Guidelines, Walking and Weakness in Children & More

SMA in Mid-March: Clinical Practice Guidelines, Walking and Weakness in Children & More

This month has seen a surge of publications focused on SMA treatments, with a trend towards optimizing various aspects of nusinersen treatment. Research has also continued in areas related to molecular mechanisms of the disease, clinical manifestations, and management...
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...