FIREFISH Parts 1 and 2: 36-month safety and efficacy of risdiplam▼ in Type 1 spinal muscular atrophy (SMA)
FIREFISH (NCT02913482) is an ongoing, multicenter, open-label study of risdiplam▼ in infants aged 1–7 months with Type 1 SMA and two SMN2 gene copies. Part 1 assessed the safety, tolerability, PK and PD of different risdiplam▼ dose levels; Part 2 assesses the safety and efficacy of the Part 1-selected dose of risdiplam▼. This poster presentation will report data from infants in Part 1 and 2 who have received risdiplam▼ treatment for 36 months.