An Open-Label Study for Previously Treated Ataluren (PTC124) Patients With Nonsense Mutation Dystrophinopathy

Trial Profile

An Open-Label Study for Previously Treated Ataluren (PTC124) Patients With Nonsense Mutation Dystrophinopathy

Active, no longer recruiting
Phase of Trial: Phase III

Latest Information Update: 27 Apr 2017

At a glance

  • Drugs Ataluren (Primary)
  • Indications Duchenne muscular dystrophy
  • Focus Adverse reactions
  • Sponsors PTC Therapeutics
  • Most Recent Events

    • 27 Apr 2017 The study is completed in Spain
    • 06 Oct 2016 Results analysing lung function data versus natural history data from a comparable non-ambulatory cohort presented as part of a company-sponsored symposium, as reported by PTC Therapeutics Media Release.
    • 06 Oct 2016 Results of a preliminary assessment (n=53) compared with history data (n=114) published in the PTC Therapeutics Media Release.
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