A Phase 3 Efficacy and Safety Study of Ataluren (PTC124) in Patients with Nonsense Mutation Dystrophinopathy

2014

This research study will test an investigational drug called ataluren. In previous studies, as well as in nonclinical models of genetic disease, ataluren has demonstrated the ability to specifically and selectively enable ribosomla read through of MRNA containing a premature stop codon, inducing production of protein that localizes to the appropriate cellular location and is functionally active. In vitro and in vivo studies have shown that ataluren can restore production of the missing dystrophin. The purpose of this study is to obtain additional information on whether the actions of ataluren can slow disease progression as assessed by tests of walking, muscle function and health related quality of life in males with DMD and to provide additional information on the safety of ataluren over 48 weeks.

Performing Research or Evaluation

Health-Related Activities

Professionals and Para-Professionals, Family Members/Caregivers, Adults with Disabilities

Other

State

N/A