An observational study in Muscular Dystrophy, Duchenne, sponsored by PTC Therapeutics. Completed at 71 sites in 16 countries. Open to participants aged 2 Years and older. Per ClinicalTrials.gov, last updated 2026-02-24.
Sponsored by PTC Therapeutics · Observational
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.
This is a multicenter, observational study of participants receiving Translarna based on inclusion of their data in a registry. This study is intended to enroll 360 participants across \~50 care centers in Europe and other regions over a period of \~ 2 years. The study population will include participants who are receiving usual care treatment with commercial supply of Translarna (or receiving care within a named participant early access program) and who provide consent. Participants will be followed for at least 5 years from their date of enrollment. Safety and efficacy data will be collected in conjunction with routine visits conducted as per usual care. Although there are no protocol-mandated procedures, it is expected that physicians and other caregivers will follow published treatment guidelines and standards of care.
473 studies on the registry are indexed under Muscular Dystrophy, Duchenne; 107 are open to participants now.
This study's enrollment of 316 is above the median of 60 across 130 observational studies indexed under Muscular Dystrophy, Duchenne.
Browse Muscular Dystrophy, Duchenne studies →PTC Therapeutics is the lead sponsor of 65 studies on the registry; 3 are open to participants now.
Of its 18 completed or terminated interventional studies of FDA-regulated products, 14 (78%) have results posted.
Counted across the registry records on this site, refreshed daily.
The target study population will include participants for whom the decision to prescribe Translarna (outside of a clinical trial) has already been made, including participants in named participant early access programs. Participants within each prescriber's practice who are or will be receiving treatment with Translarna, and who meet the eligibility criteria and provide informed consent (either by the participant or through authorisation by a legal guardian) will be invited to enroll into the study and will be followed according to the protocol.
Exclusion Criteria:
Participants with nmDMD receiving Translarna will be followed for at least 5 years from their date of enrollment, or until participant withdrawal of consent or death, whichever occurs first.
Percentage of Participants With Adverse Events
Time frame: 5 years
Prescriber and Participant Compliance With Prescribing Information According to the Approved Labelling
Time frame: 5 years
Participant Health Management Measures
Time frame: 5 years
This study is completed, as verified in Apr 2025. You cannot join it, but the record below documents what was studied.
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PTC Therapeutics