Rare Disease Day 2022.
1 week to go until Rare Disease Day 2022.
Gene Therapy Trial for GSD1a Progress.
Trials for a gene therapy treatment for GSD1a are continuing.
GSD3 Website Updates.
New research information has been added to the GSD3 website page.
Over The Wall Camp Opportunities.
Over the Wall have residential and virtual camps available in 2022.
Extension of Early Access to Medicines Scheme for trial Pompe drug.
An update on avalglucosidase alfa.