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Diaphragm Delivery of rAAV1-CMV-GAA in Pompe Disease
NCT00976352Phase 2Completed
- Sponsor
- University of Florida
- Start
- Sep 2010
- Primary completion
- Dec 2015
- Modality
- Gene Editing, Gene Silencing, Gene Addition/Replacement
- Countries
- 1
01
Overview
A Phase 2 trial of rAAV1-CMV-GAA in Pompe disease, sponsored by University of Florida. Started Sep 2010; status: completed.
02
Drugs
rAAV1-CMV-GAA
03
Conditions
Pompe disease
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