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Long-term safety and efficacy follow-up of DTX401 in adults with glycogen storage disease type Ia
NL-OMON59387Phase 2Completed
- Sponsor
- Ultragenyx Pharmaceutical Inc.
- Start
- Oct 2021
- 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 and serious adverse events. Started Oct 2021; status: completed.
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Drugs
pariglasgene brecaparvovec
03
Conditions
glycogen storage disease type 1a (GSD1a)
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