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At Week 96 participants experienced even greater reductions in daily cornstarch intake while maintaining low levels of hypoglycemia, improved levels of euglycemia and improved fasting tolerance
Phase 3 results are further supported by early data from open-label Japanese cohort (n=3) where all participants were able to eliminate daily cornstarch while maintaining or improving glycemic control
DTX401 was well tolerated with an acceptable safety profile
NOVATO, Calif., Sept. 08, 2025 (GLOBE NEWSWIRE) -- Today, Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) announced positive longer-term results from its Phase 3 study of DTX401 AAV gene therapy for the treatment of glycogen storage disease type Ia (GSDIa). Previously reported 48 Week data show that patients treated with DTX401 (n=20) experienced statistically significant and clinically meaningful reductions in daily cornstarch intake compared to placebo, while maintaining glycemic control. At Week 96, even greater reductions in cornstarch were observed with maintained low levels of hypoglycemia, improved levels of euglycemia, and improved fasting tolerance. The data were presented at the International Congress of Inborn Errors of Metabolism (ICIEM) 2025 in Kyoto, Japan.
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