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Trial intelligence / Profile preview
Long-term follow-up of DTX401 gene therapy in adults with glycogen storage disease type Ia
NL-OMON54186Phase 2Recruiting
- Sponsor
- Ultragenyx Pharmaceutical Inc.
- Start
- Oct 2021
- Modality
- Gene Therapies
- Countries
- 1
01
Overview
Phase 2, open-label, single-arm trial of pariglasgene brecaparvovec in glycogen storage disease type 1a (GSD1a), sponsored by Ultragenyx Pharmaceutical Inc. Started Oct 2021; status: recruiting.
02
Drugs
pariglasgene brecaparvovec
03
Conditions
glycogen storage disease type 1a (GSD1a)
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