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)
1. Phase 3 trial of DTX401 shows significant reduction in cornstarch intake. 2. Patients maintained glycemic control while reducing dietary carbohydrate dependence. 3. High levels of patient-reported quality of life improvements noted. 4. DTX401 demonstrated an acceptable safety profile with manageable side effects. 5. Potential to transform treatment for glycogen storage disease type Ia.