Clinical Trial Alert: Phase 2 Study of Ataluren in Children with Nonsense Mutation DMD
Researchers at Rare Disease Research, LLC are seeking boys between the ages of 6 months and 2 years who are living with Duchenne muscular dystrophy caused by a nonsense mutation (nmDMD) to participate in a phase 2 clinical trial to evaluate the safety and duration of effect of ataluren (brand name Translarna) to treat nmDMD. Ataluren . . .