Update on NICE assessment of new treatments.
An update on the appraisal of treatments for Pompe by NICE.
Moderna mRNA trial infusion for GSD1a.
First patient receives Moderna mRNA trial infusion for GSD1a.
What is a Clinical Trial?
Researchers are looking for new and improved drugs and treatments for Glycogen Storage Disease.
England’s first Rare Diseases Action Plan published.
More information about the newly published Rare Diseases Action Plan for England.
Research Study – Exercise and Pompe Disease.
Invitation to participate in research for UK adults living with Pompe.
Gene Therapy Trial for GSD1a Progress.
Trials for a gene therapy treatment for GSD1a are continuing.