Trial Profile

A confirmatory pivotal trial of GNT-0004 in Europe

Status: Planning

Phase of Trial: Phase III

Latest Information Update: 27 Jul 2026

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At a glance

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

Most Recent Events

27 Jul 2026 Planned number of patients changed from 64 to 72.
28 Jul 2025 According to a Genethon media release, Phase 3 double blind trials will begin in August and September in the UK and France
28 Jul 2025 According to a Genethon media release, based on data from Phase 1/2 study the company has received phase 3 authorizations.

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