Registry of Translarna (Ataluren) in Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)
← catalyst calendarSponsored by PTC Therapeutics (industry) · PTCT — their whole pipeline →. With The John Walton Muscular Dystrophy Research Centre (TREAT-NMD), Cooperative International Neuromuscular Research Group.
Phase
—
Status
Completed
Study type
Observational
Enrollment
316actual
Sites
71
Countries
Austria, Brazil, Czechia +13
Every value on this page is a field the sponsor filed with ClinicalTrials.gov, reproduced. Dates are their own estimates, revised as a study runs, unless the registry marks them actual. sources →
Source: ClinicalTrials.gov, retrieved 2026-08-19
Dates
each axis at the precision it was filed, with the registry's own basis| Date | Filed | Basis | What it is |
|---|---|---|---|
| Start | 2015-04-30 | actual | When the study began enrolling |
| Primary completion | 2025-03-31 | actual | The date the last participant is measured for the primary outcome — the readout window |
| Study completion | 2025-03-31 | actual | The whole study's end, after follow-up |
| First posted | 2015-02-24 | estimated | When this record first appeared on the registry |
| Results posted | — | When the sponsor posted results to the registry | |
| Record updated | 2026-02-24 | actual | The sponsor's own last edit to this record — every projection above is as current as this date |
What it studies
Condition
- Muscular Dystrophy, Duchenne
Primary outcome
what the primary-completion date above is the date OFPercentage of Participants With Adverse Events
measured 5 years
Publications
- PMID 37115359 — linked by the registry
- PMID 31997646 — linked by the registry
Linked on the registry record. A “linked by the registry” entry is NLM's own automated PubMed match, not a claim by the sponsor that the paper reports this study.
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