Last updated on July 2020

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

Are you eligible to participate in this study?

You may be eligible for this study if you meet the following criteria:

  • Age: Between 2 - 100 Years
  • Gender: Male or Female

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:

  • 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

Recruitment Status: Open

Brief Description Eligibility Contact Research Team

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