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 baseline demographics of enrolled infants with presymptomatic SMA and reports preliminary efficacy and safety data in presymptomatic infants treated with risdiplam. Bulbar function data from infants treated with risdiplam are presented for the first time.