Participants
Duchenne
All participants (N = 16) who completed the initial 24-week, phase 2 study qualified and transitioned into the LTE study. The majority of participants were White (15/16, 94%), with mean age in the LTE study being 7.9 years. Two participants enrolled when they were 4 years old. Overall, baseline characteristics between participants in the two dosage cohorts were balanced and similar to the CINRG DNHS controls (Participant baseline demographicsBMI, body mass index; CINRG, Cooperative International Neuromuscular Research Group; DNHS, Duchenne Natural History Study.