Wales Rare Diseases Action Plan.
Action Plan The Welsh Government have now published their Rare Diseases Action Plan in support of the 2021 UK Rare Disease Framework. The Wales Rare Diseases Action Plan 2022-2026 is available at the following links: English version Welsh version ...
New and Improved CureGSD1b Website.
The updated CureGSD1b website is now live.
GSD & ME website is updated.
The updated GSD & Me website is now live.
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.