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scAAV9.U7.ACCA for Exon 2 Duplication DMD
NCT04240314Phase 2Completed
- Start
- Jan 2020
- Primary completion
- Nov 2023
- Modality
- Gene Editing, Gene Silencing, RNA Therapeutics
- Countries
- 1
01
Overview
Phase 2, open-label trial of scAAV9.U7-ACCA in Duchenne muscular dystrophy (DMD). Primary endpoint: Number of Participants With Unacceptable Toxicity at 2 years. Started Jan 2020; status: completed.
02
Drugs
scAAV9.U7-ACCA
03
Conditions
Duchenne muscular dystrophy (DMD)
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