SMA is a neuromuscular disorder caused by a mutation in the SMN1 gene, which leads to a decrease in SMN protein, a protein necessary for survival of motor neurons.<unk>  is a biologic drug consisting of AAV9 virus capsids that contains a SMN1 transgene along with synthetic promoters. Upon administration, the AAV9 viral vector delivers the SMN1 transgene to the affected motor neurons, where it leads to an increase in SMN protein.
Onasemnogene abeparvovec
Voretigene neparvovec
Alipogene tiparvovec
Valoctocogene roxaparvovec
Ofranergene obadenovec
Enadenotucirev
Vibegron
Belantamab mafodotin