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completed

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

key information

study id #: NCT03508947

condition: Duchenne Muscular Dystrophy

status: completed

purpose:

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.

intervention: WVE-210201, Placebo

mechanism of action: Exon-skipping to promote dystrophin production

results: https://clinicaltrials.gov/ct2/show/results/NCT03508947

last updated: April 27, 2019

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