Clinical Trial Update: AAV9 U7snRNA Gene Therapy to Treat Boys With DMD Exon 2 Duplications. [Phase 1, Phase 2]

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Summary

Phase 1/2 clinical trial of an AAV9 U7snRNA gene therapy for Duchenne muscular dystrophy caused by exon 2 duplications has completed, with results posted.

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Diseases / indications

• Duchenne Muscular Dystrophy

Drugs mentioned

• AAV9 U7snRNA gene therapy (AAV9 U7snRNA gene therapy)

Snippet

Sponsor: Megan Waldrop | Conditions: Duchenne Muscular Dystrophy | Phase 1, Phase 2 | Status: COMPLETED | Results posted: 2025-09-11