Storage of glycogen is essential for glucose homeostasis and for energy supply during bursts of activity and sustained muscle work. We describe three siblings with profound muscle and heart glycogen ...

Fueling your morning workouts with fruit offers significant benefits. After an overnight fast, your body's energy stores are ...

Glycogen storage diseases (GSDs) constitute a group of inherited metabolic disorders that impair normal glycogen synthesis or degradation, resulting in the accumulation of structurally abnormal ...

Von Gierke disease, also known as Type I glycogen storage disease, occurs due to impaired breakdown of glycogen to glucose. Von Gierke disease is a genetic condition in which the body cannot breakdown ...

Ultragenyx is gearing up to take its glycogen storage disease (GSD) treatment to regulators next year after the gene therapy helped patients reduce their intake of cornstarch, which is currently used ...

GLYCOGEN STORAGE DISEASE TYPE III (GSD III) was diagnosed in 4 Inuit children (3 confirmed, 1 suspected case) at our institution over the last decade. This rare autosomal recessive disease, which ...

Shares of Ultragenyx Pharmaceutical RARE were up 15.5% yesterday following the completion of the rolling submission of a ...

The rare and deadly genetic liver disorder, GSD type Ia, affects children from infancy through adulthood, causing dangerously low blood sugar levels and constant dependence on glucose consumption in ...

Unexplained left ventricular hypertrophy often prompts the diagnosis of hypertrophic cardiomyopathy, a sarcomere-protein gene disorder. Because mutations in the gene for AMP-activated protein kinase γ ...

If approved, DTX401 will be the first therapy approved to treat the underlying cause of GSDIaNOVATO, Calif., Dec. 30, 2025 ...