UK Studies for Pompe disease

UK Studies for Pompe disease

*Research Update - 17/09/20

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.

Research for new therapies for Pompe disease is still a little uncertain for the immediate future, but clinical trials for Gene Therapy have begun to reopen after the programmes were paused earlier this year due to Covid-19.

We have updated the table of clinical trials (below) with the latest information provided to us, but please be aware that the situation may change as winter approaches and a second wave of Covid-19 puts NHS hospitals under strain once again.

UK Study Centres

The spreadsheet 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