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DMD is a rare, degenerative, genetic disease that results in progressive muscle loss and premature death. Affecting 1 in 5000 male births, DMD is caused by the inability to produce the dystrophin protein. There are no approved drugs available to treat DMD, although glucocorticoids show a modest effect with long-term efficacy limited by side effects. Eteplirsen is an investigational therapy designed to enable functional dystrophin production in boys who are amenable to exon 51-skipping therapy (∼13%). Twelve boys aged 7–13years with eligible genotypes were randomized 1:1:1 to 30mg/kg, 50mg/kg, or placebo. Upon completion of a 24-week double-blind, placebo-controlled study phase, all subjects were enrolled in an open-label extension and the placebo-treated subjects initiated eteplirsen treatment. The critical clinical endpoint was the change in 6-min walk test (6MWT) distance from baseline compared to the placebo/delayed-treatment cohort. Previously reported 48-week results showed a statistically significant increase in 6MWT distance for eteplirsen versus the placebo/delayed-treatment arm. At 62weeks, a significant clinical benefit of 62m was observed (p ⩽0.007) on the 6MWT for ambulatory-evaluable subjects in the combined eteplirsen-treated cohorts (n =6) versus the placebo/delayed-treatment cohort (n =4). The eteplirsen-treated cohorts showed less than a 5% decline in 6MWT distance from baseline. No clinically significant treatment-related adverse events were seen through 62weeks. Through 62weeks of treatment, eteplirsen demonstrated a significant clinical benefit on the 6MWT with no clinically significant treatment-related adverse events. Data on these outcomes will be reported through 2years of treatment. [Copyright &y& Elsevier]