Darras BT, Masson R, Mazurkiewicz-Bełdzińska M, et al. - Researchers examined the efficacy and safety of daily risdiplam in infants with type 1 spinal muscular atrophy (SMA) vs no treatment in historical controls. They carried out an open-label study of risdiplam in infants with type 1 SMA who were 1 to 7 months of age at enrollment. In total, 41 infants were recruited. Pneumonia, bronchiolitis, hypotonia, and respiratory failure were the most common serious adverse events. In this trial of infants with type 1 SMA, risdiplam resulted in larger percentages of infants meeting motor milestones and improving motor function than the percentages observed in historical cohorts. Longer and larger trials are needed to determine the long-term safety and effectiveness of risdiplam in infants with type 1 SMA.