Findacure’s Cambridge rare disease showcase.
AGSD-UK supported Findacure’s networking event on rare diseases in Cambridge, represented by Gemma Seyfang of the Pompe Support Team.
BMJ journal publishes education and practice notes on CK.
BMJ journal “Archives of Disease in Childhood” publishes education and practice notes on Creatine Kinase, including its role in signposting GSDs.
McArdle Disease Handbook reaches 30 countries.
Recent sales in Eastern Europe and the Middle East mean “The McArdle Disease Handbook” has reached patients and professionals in 30 countries.
Journal reviews progress on gene therapy for GSDs.
A paper in the journal “Human Molecular Genetics” reviews the development of gene therapy for glycogen storage diseases.
Dylan’s idea to help his friend leads to breakthroughs.
“So Chocolate Bar” – 6 years later and one boy’s idea to help his friend with GSD1b is leading to scientific breakthroughs.
Collaboration boosts prospect of GSD3 therapy.
Ultragenyx and Arcturus announce expansion of their research collaboration to develop gene therapies for rare diseases.