Safety Study of Eteplirsen to Treat Early Stage Duchenne Muscular Dystrophy
Status:
Completed
Trial end date:
2018-12-17
Target enrollment:
Participant gender:
Summary
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.