Nature13d
Two New Mutations in the Glucose-6-Phosphatase Gene Cause Glycogen Storage Disease in Hungarian Patients
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 ...
MedPage Today on MSN4d
Gene Therapy Promising for Rare Genetic Cardiomyopathy
CHICAGO -- Gene therapy for Danon disease, a rare inherited cause of hypertrophic cardiomyopathy, appeared to improve or ...
Nature8d
Gene therapy articles from across Nature Portfolio
In this mouse study, an adenine base editor shows potential as a durable therapy for glycogen storage disease type-Ia, correcting both the life-threatening lack of blood glucose control and ...
Ultragenyx Reports Third Quarter 2024 Financial Results and Corporate Update
Detailed price information for Ultragenyx Pharmaceu (RARE-Q) from The Globe and Mail including charting and trades.
Pompe Disease Pipeline Insight 2024,with Comprehensive Insights for 15+ Companies and 20+ Pipeline Drugs in the Pompe Disease Pipeline Landscape
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 ...