The drug is used to treat spinal muscular atrophy associated with a mutation in the SMN1 gene. It is administered directly to the central nervous system (CNS) using intrathecal injection. In clinical trials, the drug halted the disease progression. In around 60% of infants affected by type 1 spinal muscular atrophy, it improves motor function.
Nusinersen
Inotersen
Belinostat
Memantine
Glybuzole
Glysobuzole
Ozanimod
Siponimod