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 ...

In a global first, UConn Health, in collaboration with Connecticut Children's, has dosed a patient in a groundbreaking clinical trial testing gene editing technology to treat glycogen storage disease ...

Glycogen is a form of glucose that helps regulate your blood sugar levels. Your eating and exercise habits play a role in determining your glycogen levels. Glycogen is the stored form of a simple ...

NOVATO, Calif., Sept. 08, 2025 (GLOBE NEWSWIRE) -- Today, Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) announced positive longer-term results from its Phase 3 study of DTX401 AAV gene therapy for the ...

The brain has a hidden "sugar code" that could lead to better treatments for neurological diseases like Alzheimer’s, according to new research. A study recently published in the journal Nature ...

A new study reveals that a single gene plays a big role in how the liver stores energy, a process that's critical for overall health and for managing diseases like type 2 diabetes. The research ...

Patients with early-onset lysosomal storage diseases are ideal candidates for prenatal therapy because organ damage starts in utero. We report the safety and efficacy results of in utero ...

At Week 96 participants experienced even greater reductions in daily cornstarch intake while maintaining low levels of hypoglycemia, improved levels of euglycemia and improved fasting tolerance Phase ...