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DTX401 Gene Therapy in Glycogen Storage Disease Type Ia
DRKS00026824Phase 3Active, not recruiting
- Sponsor
- Ultragenyx Pharmaceutical Inc.
- Start
- Aug 2022
- Modality
- Gene Therapies
- Countries
- 11
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Overview
Phase 3, randomised, double-blind trial of pariglasgene brecaparvovec in glycogen storage disease type 1a (GSD1a) (2 arms), sponsored by Ultragenyx Pharmaceutical Inc. Started Aug 2022; status: active, not recruiting.
02
Drugs
pariglasgene brecaparvovec
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Conditions
glycogen storage disease type 1a (GSD1a)
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