RAINBOWFISH: A study of risdiplam▼ in infants with presymptomatic spinal muscular atrophy (SMA)
RAINBOWFISH (NCT03779334) is an open-label, single-arm, multicenter clinical study to investigate the efficacy, safety, PK and PD of risdiplam▼ in infants with genetically diagnosed SMA who are not yet presenting with symptoms. This oral presentation reports updated baseline demographics and baseline SMN protein data in enrolled infants with presymptomatic SMA. Preliminary efficacy and safety data in presymptomatic infants are presented for the first time.