Pompe disease happens when your body can't make a protein that breaks down a complex sugar, called glycogen ... as late-onset Pompe disease. This type tends to move slowly, and it doesn't usually ...

Glycogen storage disease (GSD) refers to a group of rare genetic conditions that affect how the body stores and breaks down glycogen. The most common type is type I, also called von Gierke disease.

Glycogen phosphorylase (GP) is a pivotal enzyme responsible for the catabolism of glycogen into glucose, a process intricately linked to blood glucose regulation. In the context of type 2 diabetes ...

an adeno-associated virus vector based gene therapy for the treatment of glycogen storage disease type Ia (GSDIa). Enrollment in the Phase 1/2 study is expected to begin in the first half of 2018 ...

deficiency and Glycogen Storage Disease Type Ia (GSDIa). Both diseases, due to genetically inherited inborn errors of metabolism, usually present in the neonatal period or infancy, and can have ...

Ultragenyx is gearing up to take its glycogen storage disease (GSD ... Ultragenyx’s study enrolled a total of 49 individuals with GSD Type 1a who received either the gene therapy DTX401 or ...