DMD and Duchenne muscular dystrophy: Another phase 2 study (NCT02740972) evaluating the safety and intravenous dose of viltolarsen in DMD boys aged 4–9 years showed significant drug-induced dystrophin production in both viltolarsen dose cohorts (mean [range]: 5.7% [3.2–10.3] in the 40 mg/kg group; 5.9% in the 80 mg/kg group) [41].