Outcome measures
NSAA, DMD
To measure utility, family members responding on behalf of patients completed the 15-item HUI questionnaire at screening (proxy reporting), baseline, 24 weeks, and 48 weeks (or early withdrawal). HUI responses were used to derive utility (on a scale of 0 [dead] to 1 [full health]) according to two complementary health-status classification systems: the HUI mark 3 (HUI3) and the HUI mark 2 (HUI2) [To measure motor performance, the NSAA was conducted by trained assessors at baseline and every 12 weeks for 48 weeks. The NSAA is a 17-item functional assessment scale designed for evaluating ambulant boys with DMD, where performance on tests related to motor performance (e.g., box climbing, lifting head, running) is assessed [Additional measures of ambulatory function conducted at baseline and every 12 weeks for 48 weeks included the 6MWT, RFF, and timed 10-meter walk/run (10MWR) tests. The 6MWT measures the total distance (in meters) walked by an individual on a flat hard surface within 6 min [