Zolgensma® (onasemnogene abeparvovec-xioi)
for Spinal Muscular Atrophy
Zolgensma (onasemnogene abeparvovec-xioi) is a gene therapy used to treat spinal muscular atrophy (SMA), an inherited disorder that causes muscle weakness and wasting. SMA is caused by a missing or nonfunctional SMA1 gene. Zolgensma works by delivering a new, functional copy of the human SMN gene, helping to produce the SMN protein needed to maintain healthy motor neurons and improve muscle function.
