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 ...

Discover a recent first-in-human gene therapy trial that has shown promising results in improving glucose homeostasis.

Now two years old, Mia, from Eastern Shasta County, is determined to overcome the challenges posed by Type Four Glycogen Storage Disease. Mia's mother, Amber Robertson, is working to win her ...

focusing on glycogen accumulation. This storage molecule, made up of glucose, or a simple sugar, has been found to accumulate in high levels across a variety of cancers and other diseases.

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 ...