RARE Reports Positive Long-Term Data From Metabolic Disorder Study

Key Takeaways RARE's DTX401 showed a lasting 61% mean reduction in daily cornstarch needs at week 96 in GSDIa patients.Patients reported fewer hypoglycemic events, better fasting tolerance, and higher quality of life.RARE has begun rolling BLA submission for DTX401, with completion expected later this year.Ultragenyx Pharmaceutical (RARE) reported positive longer-term data from a late-stage study of its AAV gene therapy, DTX401, for the treatment of glycogen storage disease type Ia (GSDIa). GSDIa is a rare, ...