Ultragenyx Announces Positive Longer-term Data from Phase 3 Study of DTX401 AAV Gene Therapy for the Treatment of Glycogen Storage Disease Type Ia (GSDIa)

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, ...