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Phase 3 study of DTX401 gene therapy in glycogen storage disease type Ia
jRCT2073230126Phase 3Active, not recruiting
- Sponsor
- Ultragenyx Pharmaceutical Inc.
- Start
- Aug 2024
- Modality
- Gene Therapies
01
Overview
Phase 3, open-label, single-arm trial of pariglasgene brecaparvovec in glycogen storage disease type 1a (GSD1a), sponsored by Ultragenyx Pharmaceutical Inc. Started Aug 2024; status: active, not recruiting.
02
Drugs
pariglasgene brecaparvovec
03
Conditions
glycogen storage disease type 1a (GSD1a)
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