Trial ID or NCT#



recruiting iconRECRUITING


This is an open-label study to assess the safety, tolerability, efficacy and pharmacokinetics of eteplirsen in patients with early stage Duchenne muscular dystrophy (DMD) who are amenable to exon 51 skipping.

Official Title

An Open-Label, Multi-Center Study to Evaluate the Safety, Efficacy and Tolerability of Eteplirsen in Early Stage Duchenne Muscular Dystrophy

Eligibility Criteria

Ages Eligible for Study: 4 Years to 6 Years
Sexes Eligible for Study: Male
Accepts Healthy Volunteers: No

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Bona Purse