A randomized placebo-controlled phase 3 trial of an antisense oligonucleotide, drisapersen, in duchenne muscular dystrophy

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• 48-week phase 3 RCT evaluating efficacy and safety of drisapersen 6 mg/kg/week• The pre-specified analyses did not meet statistical significance• Due to increased data variation, statistical power for 6MWD was reduced from to 53%• Evidence for a greater treatment benefit was seen in a selected population post-hoc• Most common adverse events were injection-site reactions and subclinical proteinuria

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