Safety and Tolerability of WVE-210201 in Patients With Duchenne Muscular Dystrophy
Status:
Completed
Trial end date:
2019-03-06
Target enrollment:
Participant gender:
Summary
This is a Phase 1, double-blind, placebo-controlled, single ascending dose cohort study to
evaluate the safety, tolerability, and plasma concentrations of WVE-210201 in ambulatory and
non-ambulatory male pediatric patients with DMD amenable to exon 51 skipping intervention.