Finding studies
Finding studies
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Lead
PTC Therapeutics
This study is a Phase 2b, multicenter, randomized, double-blind, placebo-controlled, dose-ranging, efficacy and safety study, designed to document the clinical benefit of ataluren when administered as therapy of patients with DMD/BMD due to a nonsense mutation (premature stop codon) in the dystrophin gene.
Age
5–any
Sex
MALE
Healthy volunteers
Not accepted