SMN1 and spinal muscular atrophy, type 1: Further evidence on FIREFISH Part 1 was published by Baranello et al., where they evaluated the safety, PK, PD, and dose selection for risdiplam among infants aged 1–7 months with genetically confirmed Type 1 SMA, two SMN2 copies, and who had not previously received other SMN-targeted or any other gene therapy.