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DTX401 gene therapy in adults with glycogen storage disease type Ia
NL-OMON48684Phase 2Completed
- Sponsor
- Ultragenyx Pharmaceutical Inc.
- Start
- Oct 2019
- Modality
- Gene Therapies
- Countries
- 1
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Overview
Phase 2, open-label, single-arm trial of pariglasgene brecaparvovec in glycogen storage disease type 1a (GSD1a), sponsored by Ultragenyx Pharmaceutical Inc. Primary endpoint: Incidence of adverse events including dose-limiting toxicities. Started Oct 2019; status: completed.
02
Drugs
pariglasgene brecaparvovec
03
Conditions
glycogen storage disease type 1a (GSD1a)
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