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.

McArdle disease, also known as glycogen storage disease type V, is an inherited metabolic disorder marked by a deficiency of myophosphorylase, the enzyme essential for the breakdown of glycogen in ...

Mumbai: A one-year-old boy suffering from Glycogen Storage Disease Type 1a (GSD Ia), a rare inherited metabolic liver disorder, has been successfully diagnosed and treated, offering a major ...

NOVATO, Calif., July 26, 2018 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare ...

River Pugh, a three-year-old boy from Indiana, is fighting to survive Pompe Disease, a rare genetic disease that causes ...

Treatment with DTX401 resulted in a statistically significant reduction in daily cornstarch intake at Week 48 (p<0.0001) with maintenance of glucose control Company will host investor call today at ...

Introduction: Glycogen storage disease Type III (GSD III) is an autosomal recessive disease caused by the deficiency of glycogen debranching enzyme, encoded by the AGL gene. Two clinical types of the ...