Glycogen storage disease type 1a (von Gierke disease, GSD1a), one of the most common types of glycogen storage diseases, is caused by the deficiency of microsomal glucose-6-phosphatase (G6Pase ...
glycogen storage diseases, and progressive familial intrahepatic cholestasis (PFIC) Prevention and Management Adopting a healthy lifestyle is crucial to reduce the risk of liver disease in adulthood.
Discover a recent first-in-human gene therapy trial that has shown promising results in improving glucose homeostasis.
Gerty Theresa Cori (1896-1957) was a trailblazing biochemist whose work fundamentally reformed our understanding of how cellular energy is stored and ...
1). Preclinical programs include wholly owned mRNA therapeutics for cystic fibrosis and influenza, along with partnered programs in glycogen storage disease type 3, hepatitis B, and non-alcoholic ...