Registry of Translarna (Ataluren) in Nonsense Mutation Duchenne Muscular Dystrophy
Status:
Recruiting
Trial end date:
0000-00-00
Target enrollment:
Participant gender:
Summary
This study is being performed as a post-approval safety study (PASS), per the
Pharmacovigilance Risk Assessment Committee (PRAC) of the European Medicines Agency (EMA),
to gather data on Translarna (ataluren) safety, effectiveness, and prescription patterns in
routine clinical practice.
Phase:
N/A
Details
Lead Sponsor:
PTC Therapeutics
Collaborators:
Cooperative International Neuromuscular Research Group (CINRG) TREAT-NMD