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GSDIa Disease Monitoring Program
2023-510219-20-00Phase 4Authorised
- Sponsor
- Ultragenyx Pharmaceutical Inc.
- Start
- Sep 2024
- Modality
- Gene Therapies
- Countries
- 10
01
Overview
Phase 4, open-label trial of pariglasgene brecaparvovec in glycogen storage disease type 1a (GSD1a) (2 arms), sponsored by Ultragenyx Pharmaceutical Inc. Primary endpoints: SAEs related to DTX401, Adverse events of special interest for AAV therapies and 3 more. Planned start Sep 2024; status: authorised.
02
Drugs
pariglasgene brecaparvovec
03
Conditions
glycogen storage disease type 1a (GSD1a)
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