A Phase 3 Efficacy and Safety Study of Ataluren (PTC124) In Patients with Nonsense Mutation Dystrophinopathy

Trial Profile

A Phase 3 Efficacy and Safety Study of Ataluren (PTC124) In Patients with Nonsense Mutation Dystrophinopathy

Completed
Phase of Trial: Phase III

Latest Information Update: 30 Sep 2017

At a glance

  • Drugs Ataluren (Primary)
  • Indications Duchenne muscular dystrophy; Muscular dystrophies
  • Focus Registrational; Therapeutic Use
  • Acronyms ACT DMD
  • Sponsors PTC Therapeutics
  • Most Recent Events

    • 18 Jul 2017 According to a PTC Therapeutics media release, results from this trial published in The Lancet.
    • 17 Jul 2017 Results assessing the efficacy and safety of ataluren in ambulatory boys with nonsense mutation Duchenne muscular dystrophy (DMD) published in The Lancet.
    • 06 Jun 2017 According to a PTC Therapeutics media release, the U.S. Food and Drug Administration (FDA) has notified the company of the tentative scheduling of a Peripheral and Central Nervous Systems Drugs Advisory Committee meeting on September 28, 2017 to review the new drug application (NDA) for ataluren (Translarna).
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