<unk> , sold under the brand name Zolgensma, is a gene therapy medication used to treat spinal muscular atrophy (SMA). It is used as a one-time infusion into a vein.<unk>  works by providing a new copy of the gene that makes the human SMN protein. The treatment must be accompanied by a course of corticosteroids of at least two months. Common side effects include vomiting and increased liver enzymes.<unk>  was first approved for medical use in the United States in 2019 as a treatment for children less than two years old. It was later approved in other jurisdictions with similar scope. The approval scope in certain jurisdictions, including the European Union and Canada, is somewhat different.
Onasemnogene abeparvovec
Voretigene neparvovec
Alipogene tiparvovec
Valoctocogene roxaparvovec
Ofranergene obadenovec
Enadenotucirev
Vibegron
Belantamab mafodotin