RAINBOWFISH: Preliminary efficacy and safety data in risdiplam▼-treated 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 symptoms. This poster describes the study design and reports the baseline data of the first 18 infants enrolled. It also reports exploratory efficacy data from the first 7 infants to be treated with risdiplam▼ for 12 months.