UK Studies for Pompe disease
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, ClinicalTrials.gov 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 firstname.lastname@example.org.
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 Trials.gov
EMA European Clinical Trials Register
WHO International Clinical Trials Register
Notes from SSIEM 2019, Rotterdam, 3-5 September 2019
Allan Muir, Pompe UK,
Allan Muir attended the 2019 symposium of the Society for Study of Inborn Errors of Metabolism (SSIEM) on behalf of the UK LSD Collaborative. It was a fantastic networking event with mainly specialists in metabolic medicine and industry representatives in attendance. The programme featured an abundance of oral and poster presentations concerning Lysosomal Diseases, many including Pompe disease and featured current therapies in the clinic and under development.
Read Allan's visit report here