A new paper reports on the amelioration of disease through delivery of gene therapy in the mouse model of McArdle disease.
AGSD-UK supported Findacure’s networking event on rare diseases in Cambridge, represented by Gemma Seyfang of the Pompe Support Team.
BMJ journal “Archives of Disease in Childhood” publishes education and practice notes on Creatine Kinase, including its role in signposting GSDs.
Recent sales in Eastern Europe and the Middle East mean “The McArdle Disease Handbook” has reached patients and professionals in 30 countries.
A paper in the journal “Human Molecular Genetics” reviews the development of gene therapy for glycogen storage diseases.
“So Chocolate Bar” – 6 years later and one boy’s idea to help his friend with GSD1b is leading to scientific breakthroughs.