Open-label Study of WVE-N531 in Patients With Duchenne Muscular Dystrophy
Status:
Recruiting
Trial end date:
2022-09-01
Target enrollment:
Participant gender:
Summary
This is a Phase 1b/2a open-label study to evaluate the safety, tolerability, pharmacokinetic
(PK), pharmacodynamic (PD), and clinical effects of intravenous (IV) WVE-N531 in patients
with Duchenne muscular dystrophy (DMD). To participate in the study, patients must have a
documented mutation of the DMD gene that is amenable to exon 53 skipping intervention