RARE — Phase 3 DTX401 trial completion in GSDIa
Feb 1, 2026, 5:00:00 AM UTC
Summary
Ultragenyx Pharmaceutical is expected to complete its Phase 3 clinical trial of DTX401, an investigational gene therapy for Glycogen Storage Disease Type Ia (GSDIa), on February 1, 2026. The study, involving 49 participants, evaluates the efficacy and safety of DTX401 in patients aged 8 and older. The trial includes a crossover design and a long-term follow-up program. DTX401 is an AAV8-mediated gene therapy candidate targeting GSDIa, a rare metabolic disorder.
Company
ULTRAGENYX PHARMACEUTICAL INC (RARE)NASDAQ — Healthcare: Manufacturing, Biotechnology: Pharmaceutical Preparations
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