April 23, 2018 Ultragenyx Announces Filing and FDA Clearance of an Investigational New Drug Application for DTX401, a Gene Therapy for the Treatment
of Glycogen Storage Disease Type Ia
These findings impact our understanding
of glycogen storage disease XV where patients lack glycogenin - 1 and accumulate muscle glycogen.
Not exact matches
Glycogen storage disease VII, PFK deficiency is an inherited metabolic disorder affecting several breeds
of dog.
Mass lesions within the liver are often identified on ultrasound examination and can represent an area
of liver healing (or regenerative nodule in a damaged region), a change in tissue content caused by
storage of glycogen (starch, common in dogs with Cushing's
Disease described below), infections (abscesses), biliary cysts, chronic fibrous tissue secondary to chronic hepatitis, or benign or malignant tumors.
Pyruvate kinase (PK) deficiency has been found in the breed and
glycogen storage disease type IV, an inherited abnormality
of glucose metabolism, is also seen in Norwegian Forest cats.