Study design and outcomes
risdiplam
Patients were stratified by age (2–5, 6–11, 12–17, and 18–25 years) and randomized 2:1 with concealed allocation to receive either risdiplam or placebo daily for 12 months. The risdiplam dose was 0.25 mg/kg for patients weighing < 20 kg, and 5 mg for patients weighing ≥ 20 kg [The 24-month exploratory objectives and outcomes included the efficacy of risdiplam treatment with regard to motor function (as measured by MFM32 [Safety was assessed throughout the study by monitoring and recording AEs, including serious AEs (SAEs), laboratory assessments, electrocardiograms, vital signs, and ophthalmologic, neurologic, and anthropometric examinations.