Long-Term Observational Study of Translarna Safety and Effectiveness in Usual Care

This is a Phase IV, multicenter, observational study being performed as a post-approval safety study (PASS) to assess the long-term clinical benefit of Translarna (Ataluren) when used in routine clinical practice. Ataluren which is being marketed as Translarna for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD) in patients aged 2 years and older. Enrolled patients will be followed for at least 5 years from the date of enrollment, or until withdrawal from the study. To learn more about the study eligibility and participating sites please visit: https://clinicaltrials.gov/ct2/show/NCT02369731.