Ultragenyx Pharmaceutical Says Glycogen Storage Disease Treatment Met Endpoints

Ultragenyx Pharmaceutical on Thursday said its potential treatment for glycogen storage disease type 1a met multiple endpoints in a clinical study.

The Novato, Calif.-based biopharmaceutical company said its gene therapy DTX401 had resulted in a statistically significant and clinically meaningful reduction in daily cornstarch intake after 48 weeks, which was the study's primary endpoint.