A Study to Assess Dystrophin Levels in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)
Status:
Completed
Trial end date:
2020-10-30
Target enrollment:
Participant gender:
Summary
This study is designed to evaluate the ability of ataluren to increase dystrophin protein
levels in muscle cells of participants with nmDMD. The study will evaluate the levels of
dystrophin before and after 40 weeks of ataluren therapy using muscle biopsies and 2
validated assay methods, electrochemiluminescence (ECL) and immunohistochemistry.