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A Phase 2b Efficacy and Safety Study of PTC124 in Subjects With Nonsense-Mutation-Mediated Duchenne Muscular Dystrophy and Becker Muscular Dystrophy

Trial Profile

A Phase 2b Efficacy and Safety Study of PTC124 in Subjects With Nonsense-Mutation-Mediated Duchenne Muscular Dystrophy and Becker Muscular Dystrophy

Status: Completed
Phase of Trial: Phase II

Latest Information Update: 25 Jul 2019

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At a glance

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

    • 31 Aug 2018 Biomarkers information updated
    • 14 Mar 2016 According to a PTC Therapeutics media release, the company intends to submit results of a recently completed Phase 3 study (CTP 237860) for review by Health Canada as part of the New Drug Submission (NDS) for Translarna (ataluren) to treat nonsense mutation Duchenne muscular dystrophy (nmDMD). In order to submit this additional data for review, the company will withdraw current NDS and resubmit the NDS with phase 3 results. As a result, the company no longer expects review of the NDS in 1H 2016.
    • 18 Sep 2015 Health Canada has initiated their review of the company's New Drug Submission for Translarna (ataluren) to treat nonsense mutation dystrophinopathy. The NDS will be reviewed with advance consideration under the Health Canada policy of Notice of Compliance with Conditions (NOC/c), according to a PTC Therapeutics media release.
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