Preclinical and early clinical trial data strongly suggest that early treatment has the potential to maximize treatment benefit in children with Duchenne muscular dystrophy (DMD). With the historic average range of diagnosis between the ages of 3-5 years, the magnitude of change in development as a result of disease natural history is not as well defined as in the older cohorts. This study aims to examine the utility of the Bayley-III, NSAA, and 100m for use in the very young DMD cohort, confirm differences in motor performance between young boys with DMD and typically developing peers and document natural history data and reference values in young boys with DMD.

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