Despite decades of use in Duchenne muscular dystrophy (DMD), the optimal corticosteroid regimen for treatment remains unresolved. Daily use of prednisone or deflazacort delays loss of ambulation (LOA) but is associated with significant side effects. We evaluated the effect of an intermittent weekend dosing regimen, intended to minimize side effects, on LOA in a retrospective/observational study cohort within the United Dystrophinopathy Project (UDP), which enrolled, genotyped, and phenotyped 682 DMD subjects between 2003 and 2011.

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