Presymptomatic treatment of spinal muscular atrophy with risdiplam leads to improved functional outcomes

1. In this single-group trial, among infants predicted to develop type 1 spinal muscular atrophy (SMA) who were treated presymptomatically with risdiplam within the first six weeks after birth, most had the ability to sit up within 12 months, which would not be possible for most untreated infants. 2. These infants also survived without assisted […]