“So Chocolate Bar” – 6 years later and one boy’s idea to help his friend with GSD1b is leading to scientific breakthroughs.
Ultragenyx and Arcturus announce expansion of their research collaboration to develop gene therapies for rare diseases.
Twin boys born with GSD1a and 8 year old big sister Amy organised a school Fun Run to help fund research.
In May AGSD-UK facilitated a meeting of UK metabolic specialists to discuss guidelines for management of hepatic GSDs.
Dr David Weinstein reports rewarding preliminary results from the first ever gene therapy trial in a Glycogen Storage Disease.