Safety and Tolerability of WVE-210201 in Patients With Duchenne Muscular Dystrophy

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.

Timeline

Start

2018-01-24

Primary completion

2019-03-06

Completion

2019-03-06

Drugs

Indications