FDA approves treatment for GSD1a in the U.S.
The U.S. Food and Drug Administration (FDA) has approved DTX401, a gene therapy developed by Ultragenyx for the treatment of GSDIa. Find out more here:
Update on Sanofi supply issues.
Sanofi have provided the following information on the supply issues affecting their Pompe therapies.
Please remember our AGSD-UK advisors are here to listen and provide support for those affected by this uncertainty. Just contact info@agsd.org.uk
McArdle walking courses 2026
Just two months to go until the start of the 2026 McArdle walking courses. This year to be held in the beautiful Pembrokeshire National Park in south west Wales.
Booking now open for our Main Event 4th-5th July Loughborough.
Don’t miss the chance to share support, find out about latest developments and try new activities -you’ll get a warm welcome in a great venue and crèche places are available.
April 15th is International Pompe Day.
To help raise awareness Emmie has been talking about daughter Arla and the family’s experience on infant onset Pompe. You can find out more here.
15th April is International Pompe Day.
Find out more about what a typical day might look like for community members affected by Pompe and what they find most helpful.