Registry of Translarna (Ataluren) in Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)
About the study
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.
Who can take part
You may be eligible to participate in the study if you meet the following criteria:
INCLUSION CRITERIA
Inclusion Criteria:
- Receiving or will be receiving usual care treatment with commercial supply of Translarna (or receiving care within a named participant early access program)
- Willing to provide written informed consent to allow the study data collection procedures (either by the participant or through authorisation by a legal guardian)
EXCLUSION CRITERIA
Exclusion Criteria:
Participants 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
Study Locations
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How to Apply
Contact the study center to learn if this study is a good match for you.
Study’s details
Contition
Muscular Dystrophy, Duchenne
Age (in years)
2+
Participants needed
316
Est. Completion Date
May 30, 2025
Treatment type
Observational [Patient Registry]
Sponsor
PTC Therapeutics
ClinicalTrials.gov identifier
NCT02369731
Study number
PTC124-GD-025o-DMD
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