Validation of a prognostic score for changes in six-minute walk distance (6MWD) in patients with Duchenne muscular dystrophy (DMD)

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Drug efficacy trials in DMD have been clouded by variation in rates of change in ambulatory function. We previously reported a prognostic score that explained 60% of variation in 1-year 6MWD changes, and significantly improved upon prognosis from baseline age, 6MWD and steroid use by also incorporating timed function tests, height and weight. This score was developed using natural history data from Universitaire ziekenhuizen Leuven. Here we report the validation of this score using 48-week placebo arm data from the Phase 3 clinical trial of tadalafil in DMD.

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