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

NCT02369731 · Status: COMPLETED · Type: OBSERVATIONAL · Enrollment: 316

Last updated 2026-02-24

No results posted yet for this study

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.

Conditions

  • Muscular Dystrophy, Duchenne

Sponsors & Collaborators

  • The John Walton Muscular Dystrophy Research Centre (TREAT-NMD)

    collaborator UNKNOWN

  • Cooperative International Neuromuscular Research Group

    collaborator NETWORK

  • PTC Therapeutics

    lead INDUSTRY

Principal Investigators

  • Shelley Johnson · PTC Therapeutics

Eligibility

Min Age
2 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2015-04-30
Primary Completion
2025-03-31
Completion
2025-03-31

Countries

  • Austria
  • Brazil
  • Czechia
  • France
  • Germany
  • Greece
  • Hungary
  • Israel
  • Italy
  • Latvia
  • Norway
  • Portugal
  • Romania
  • Slovenia
  • Sweden
  • United Kingdom

Study Locations

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Entities

Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT02369731 on ClinicalTrials.gov