Description
Risdiplam (RG7916, RG-7916, RO7034067) is a highly potent, selective, orally activeSMN2 splicing modifier for treatment ofspinal muscular atrophy (SMA); RG7916 is undergoing clinical trials across the spectrum of spinal muscular atrophy, where it has shown promising early results. Spinal Muscular Atrophy (SMA) Phase 2 Discontinued