Novartis is a healthcare and pharmaceutical company based in Basel, Switzerland, incorporated in 1996.
ZOLGENSMA® (onasemnogene abeparvovec-xioi) is a gene therapy approved by the US Food and Drug Administration (FDA) for children less than 2 years old with spinal muscular atrophy (SMA).
ZOLGENSMA is given as a one-time infusion into a vein.
ZOLGENSMA was not evaluated in patients with advanced SMA.