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GNT0004 microdystrophin gene therapy trial in ambulant boys with Duchenne muscular dystrophy
2023-505187-11-00Phase 2Authorised
- Sponsor
- Généthon
- Start
- Mar 2021
- Modality
- AAV Vectors → Viral Vectors → Gene Addition/Replacement → Gene Therapies
- Countries
- 4
01
Overview
Phase 2, randomised, double-blind trial of GNT0004 in Duchenne muscular dystrophy (DMD) (4 arms), sponsored by Généthon. Primary endpoint: North Star Ambulatory Assessment raw total score change from baseline versus placebo. Planned start Mar 2021; status: authorised.
02
Drugs
GNT0004
03
Conditions
Duchenne muscular dystrophy (DMD)
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