Efficacy outcomes
DMD
Timed function tests were used to indicate DMD progression. For the primary efficacy endpoint (TTSTAND) participants who received viltolarsen showed stabilization of motor function over the first two years and a significant slowing of motor function loss over the following two years, whereas a more significant decline was observed in the CINRG DNHS comparator group over the entire 4-year period (Timed Function Tests: Change From Baseline vs Natural History Controls for TTSTAND. Similarly, the change from baseline for TTRW showed stabilization of motor function over the first two years and significant slowing of motor function loss over the following two years for viltolarsen-treated participants compared with the CINRG DNHS comparator group (Timed Function Tests: Change From Baseline vs Natural History for TTRW and TTCLIMB. A. TTRW change from baseline vs natural history controls. B. TTCLIMB change from baseline vs natural history controls. 