Search Results for: Zolgensma

Zolgensma (AVXS-101)

Zolgensma, previously knowns as AVXS-101, is the proprietary gene therapy candidate from AveXis designed to treat spinal muscular atrophy (SMA). How Zolgensma works SMA is caused by a mutation in the ... Read more

What is Spinal Muscular Atrophy?

Spinal muscular atrophy (SMA) is an autosomal recessive neurodegenerative disease, and the most common cause of mortality in infants linked to a genetic mutation. It affects 1 in between 8,000 ... Read more