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 ...

Detailed price information for Ultragenyx Pharmaceu (RARE-Q) from The Globe and Mail including charting and trades.

Background: Glycogen storage disease (GSD), characterized by defects in carbohydrate metabolism, may result in left ventricular hypertrophy (GSD-CM) that mimics primary hypertrophic cardiomyopathy ...

The "Pompe Disease - Pipeline Insight, 2024" clinical trials has been added to ResearchAndMarkets.com's offering.The report provides comprehensive insights about 15+ companies and 20+ pipeline drugs ...

Finally, I present a rough plan to start a position in RARE. I will also point out that Ultragenyx's pipeline is filled with ...

CHICAGO -- Gene therapy for Danon disease, a rare inherited cause of hypertrophic cardiomyopathy, appeared to improve or ...

Ex vivo precision-cut liver slices allow glucose production quantification to model glycogen storage disease and inborn errors of metabolism. This Perspective analyzes the most common maturation ...

Beam Therapeutics Inc (NASDAQ:BEAM) has received US IND clearance for their second in-vivo program, BEAM-301, for glycogen ...

Initial Clinical Data for BEAM-101 and Preclinical Non-human Primate Data for ESCAPE Accepted for Presentation at American Society of Hematology ...

Wilson’s Disease and glycogen storage disease (GSD) type 1. The delivery method is again an LNP using a GalNAc targeting ligand, which Duffield said has been “derisked and validated in humans ...