Overview

A Study to Assess Dystrophin Levels in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD) Who Have Been Treated With Ataluren

Status:
Completed
Trial end date:
2019-05-24
Target enrollment:
Participant gender:
Summary
This study is designed to generate additional data on the effect of ataluren for producing dystrophin protein in nonsense mutation nmDMD participants. This study will evaluate dystrophin levels from participants with nmDMD who currently have been receiving ataluren for greater than or equal to (>=) 9 months. The study will have a single visit (Visit 1).
Phase:
Phase 2
Details
Lead Sponsor:
PTC Therapeutics