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.
Extension of Early Access to Medicines Scheme for trial Pompe drug.
An update on avalglucosidase alfa.
CureGSD1b Research Ready Data Collection Plan.
The CureGSD1b Research Alliance is working extensively on a full understanding of the research landscape related to GSD1b at the moment.
Progress on NICE assessment of new treatments.
An update on the appraisal of treatments for Pompe by NICE.
Lived experience advisory panel.
Could you represent the rare experience on this new venture from The Patient Association?