Registry of Translarna (Ataluren) in Nonsense Mutation Duchenne Muscular Dystrophy

• Status: Recruiting
• Trial end date: 0000-00-00
• Target enrollment: 200
• Participant gender: Both

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
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: PTC Therapeutics
• Collaborators: Cooperative International Neuromuscular Research Group (CINRG); TREAT-NMD
• Last Updated: 2016-02-04

Criteria

Inclusion Criteria:

• Receiving or will be receiving usual care treatment with commercial supply of Translarna (or receiving care within a named patient early access program)

• Willing to provide written informed consent to allow the study data collection procedures (either by the patient or through authorisation by a legal guardian)

Exclusion Criteria:

• Patients who are receiving ataluren or placebo in a blinded, randomized clinical trial, or ataluren in any other ataluren clinical trial or cohort early access program that prevents participation in this study