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 ...
CHICAGO -- Gene therapy for Danon disease, a rare inherited cause of hypertrophic cardiomyopathy, appeared to improve or ...
Detailed price information for Ultragenyx Pharmaceu (RARE-Q) from The Globe and Mail including charting and trades.