Glycogen Storage Diseases (GSDs) are a group of inherited metabolic disorders that result from deficiencies in enzymes responsible for the synthesis or breakdown of glycogen, a stored form of glucose.

The company has completed a Phase 3 study around its candidate DTX401 to treat Glycogen storage disease type I or GSD1a. This is a rare, inherited disorder that inhibits the proper breakdown of ...

In 2024, Ultragenyx announced that the phase III GlucoGene study (NCT05139316), evaluating its investigational AAV8 gene therapy, DTX401, to treat glycogen storage disease type Ia patients aged ...

along with candidates for glycogen storage disease 1b (GSD1b) and retinitis pigmentosa. Data reported at the European Society of Gene and Cell Therapy Congress last October showed that it was ...