Study of Eteplirsen in Young Patients With DMD Amenable to Exon 51 Skipping
Status:
Completed
Trial end date:
2021-03-10
Target enrollment:
Participant gender:
Summary
This is a multicenter, open-label, dose-escalation study to evaluate the safety,
tolerability, PK, and efficacy of once-weekly IV infusions of eteplirsen in approximately 12
male patients, ages 6 months to 48 months (inclusive), who have genotypically confirmed DMD
with a deletion mutation amenable to exon 51 skipping.