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An Open-Label, Multi-Center, 48-Week Study with Concurrent Untreated Control Arm to Evaluate the Efficacy and Safety of Eteplirsen in Duchenne Muscular Dystrophy

Principal Investigator:
Kathryn
Wagner

This is an open-label, Phase 3, multi-center, 48-week study to evaluate the efficacy and safety of eteplirsen in patients with genotypically confirmed Duchenne muscular dystrophy (DMD) with genetic deletions amenable to correction by exon 51 skipping (treated group), with a concurrent control arm of DMD patients not amenable to exon 51 skipping (untreated group).

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