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Phase 3 study of pariglasgene brecaparvovec in glycogen storage disease type Ia
2023-508750-25-00Phase 3Completed
- Sponsor
- Ultragenyx Pharmaceutical Inc.
- Start
- Jan 2022
- Modality
- Gene Therapies
- Countries
- 9
01
Overview
Phase 3, randomised, double-blind trial in glycogen storage disease type 1a (GSD1a) comparing Pariglasgene brecaparvovec arm with Placebo arm (2 arms). Started Jan 2022; status: completed.
02
Drugs
pariglasgene brecaparvovec
03
Conditions
glycogen storage disease type 1a (GSD1a)
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