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Trial Profile

A Phase 3, Randomized, Double-blind, Placebo-controlled Efficacy and Safety Study of Ataluren in Patients With Nonsense Mutation Duchenne Muscular Dystrophy and Open-Label Extension

Status: Completed
Phase of Trial: Phase III

Latest Information Update: 05 Nov 2024

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At a glance

  • Drugs Ataluren (Primary)
  • Indications Duchenne muscular dystrophy
  • Focus Registrational; Therapeutic Use
  • Acronyms Study 041
  • Sponsors PTC Therapeutics
  • Most Recent Events

    • 30 Oct 2024 According to PTC Therapeutics media release, the U.S. FDA has accepted for review the resubmission of the NDA for Translarna for the treatment of nmDMD based on the data from this and STRIDE studies.
    • 01 Jul 2024 According to PTC Therapeutics media release, the company has resubmitted the NDA for Translarna for the treatment of nmDMD in July 2024.
    • 25 Apr 2024 According to PTC Therapeutics media release, Based on FDA feedback, PTC plans to resubmit the NDA for Translarna for the treatment of nmDMD in mid-2024.

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