Ultragenyx releases long-term phase 3 data on gene therapy for metabolic disorder

9 hours ago 1

Ultragenyx Pharmaceutical (NASDAQ:RARE) said that week 96 phase 3 data of its gene therapy DTX401 for glycogen storage disease type Ia (GSDIa) found that greater reductions in cornstarch were observed with low levels of hypoglycemia compared to week 48 data.