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

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Trial Profile

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

Status: Completed
Phase of Trial: Phase III

Latest Information Update: 04 Jun 2024

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At a glance

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

Most Recent Events

  • 18 Apr 2024 Results investigating the effect of ataluren plus standard of care (SoC) on age presented at the 76th Annual Meeting of the American Academy of Neurology 2024
  • 22 Apr 2021 Results presented at the 73rd Annual Meeting of the American Academy of Neurology
  • 28 Jun 2019 According to a PTC Therapeutics media release, CHMP adopted a negative opinion of the extension, PTC was informed by EMA representatives that the European Public Assessment Report (EPAR) will be updated to clarify that patients who start Translarna while ambulatory are not required to discontinue treatment after loss of ambulation. This extension was based on study 019 and 025.

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