UK Studies for Pompe disease

Posted On: 08/05/2020

There are a growing number of clinical studies for next-generation Enzyme Replacement Therapies, Gene Therapies. These are usually reported on US, EU and WHO websites, but we have selected those with UK study centres and will update our list to keep it relevant.

To find out where current UK clinical trials are taking place, and their criteria for taking part, download this spreadsheet:

UK Study Centres

The spreadsheet above contains links to the studies on the NIH register of clinical trials, and the status of studies in the UK. We will update the spreadsheet as new studies are introduced and as more information becomes available.

If you are interested in joining one of the studies, you should first discuss it with your consultant at the Highly Specialised LSD Centre you attend. You should be able to transfer to another centre if yours is not offering the trial you would like to join.

If you can’t open the spreadsheet, please contact the Pompe Support Network at

To check centres in other regions you can click on the links in our spreadsheet or search through these international registries of clinical studies:

NIH Clinical

EMA European Clinical Trials Register

WHO International Clinical Trials Register