Drug treatment for spinal muscular atrophy types II and III

Review of 10 RCTs (n=717) found that nusinersen improves motor function in spinal muscular atrophy (SMA) type II. Creatine, gabapentin, hydroxyurea, phenylbutyrate, valproic acid probably have no clinically important effect on motor function in SMA types II or III (or both).


Cochrane Database of Systematic Reviews