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.
Amy’s school Fun Run for her brother Eric.
Twin boys born with GSD1a and 8 year old big sister Amy organised a school Fun Run to help fund research.
AGSD-UK hosts workshop to develop hepatic guidelines
In May AGSD-UK facilitated a meeting of UK metabolic specialists to discuss guidelines for management of hepatic GSDs.
Promising initial results in gene therapy trial
Dr David Weinstein reports rewarding preliminary results from the first ever gene therapy trial in a Glycogen Storage Disease.