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A confirmatory pivotal trial of GNT-0004 in Europe

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A confirmatory pivotal trial of GNT-0004 in Europe

Status: Planning
Phase of Trial: Phase III

Latest Information Update: 26 Nov 2024

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At a glance

  • Drugs GNT 0004 (Primary)
  • Indications Duchenne muscular dystrophy
  • Focus Registrational; Therapeutic Use

    • Most Recent Events

    • 26 Nov 2024 New trial record
    • 19 Nov 2024 According to a Genethon media release, based on the positive results of the dose escalation part of all-in-one phase 1/2/3 study, the company aims to start this confirmatory (pivotal) phase in more than 60 children in Europe in Q2 2025, followed by the US.

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