
England’s first Rare Diseases Action Plan published.
More information about the newly published Rare Diseases Action Plan for England.
More information about the newly published Rare Diseases Action Plan for England.
Sioned Williams celebrates Rare Disease Day with a short video talk about her life and career with McArdle’s, and an appeal via JustGiving.
Invitation to participate in research for UK adults living with Pompe.
1 week to go until Rare Disease Day 2022.
Trials for a gene therapy treatment for GSD1a are continuing.
New research information has been added to the GSD3 website page.