In three clinical studies with RG7916: SUNFISH (SMA 2 and SMA 3), FIREFISH (SMA 1) and JEWELFISH (pre-treated patients with SMA 2 and SMA 3), Risdiplam showed a significant clinical benefit in infantile SMA, less pronounced in later-onset SMA [10–13]. Here, SMN1 is linked to proximal spinal muscular atrophy.