<unk>  has been developed to treat spinal muscular atrophy, a disease linked to a mutation in the SMN1 gene on chromosome 5q and diagnosed predominantly in young children that causes progressive loss of muscle function and frequently death. The medication is administered as an intravenous infusion. The treatment is approved in the United States and certain other countries for use in children with SMA up to the age of two, including at the presymptomatic stage of the disease. In the European Union and Canada, it is indicated for the treatment of patients with SMA who either have a clinical diagnosis of SMA type 1 or have up to three copies of the SMN2 gene. The medication is used with corticosteroids in an effort to protect the liver. While marketed as a one-time treatment for SMA, it is unknown how long the<unk> -delivered transgene will persist in people. Since motor neurons do not divide, it is expected that the transgene may have long-term stability.
Onasemnogene abeparvovec
Voretigene neparvovec
Alipogene tiparvovec
Valoctocogene roxaparvovec
Ofranergene obadenovec
Enadenotucirev
Vibegron
Belantamab mafodotin