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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

Status: Completed
Phase of Trial: Phase III

Latest Information Update: 25 Jul 2019

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At a glance

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

    • 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.
    • 28 Jun 2019 According to a PTC Therapeutics media release, the company announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has completed their review of a proposed indication extension of Translarna (ataluren) for the treatment of patients with nonsense mutation Duchenne muscular dystrophy (nmDMD) who are non-ambulatory.
    • 09 Apr 2019 Status changed from active, no longer recruiting to completed.
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