Would you like to help research into Pompe disease?

Would you like to help research into Pompe disease?

Posted On: 16/02/2021 Recently Posted!

If you have Late Onset Pompe disease and are not already taking part in a clinical trial for Pompe, a research team at a Newcastle study would be interested to take a blood sample from you to help them understand how antibodies in patients may affect a particular gene therapy.

Would you like to help research into Pompe disease?

Study title: An AAV8 Neutralizing Antibody Seroprevalence Study in Subjects with Late Onset Pompe Disease

Location: The John Walton Muscular Dystrophy Research Centre, International Centre for Life, Newcastle upon Tyne

If you have Late Onset Pompe disease and are not already taking part in a clinical trial for Pompe, a research team at a Newcastle study would be interested to take a blood sample from you to help them understand how antibodies in patients may affect a particular gene therapy.

This study will involve one visit to the Centre for Life in Newcastle to meet the medical staff. They will give you an explanation of the study and you’ll have an opportunity to ask any questions prior to giving your consent and having a blood sample taken.

You will receive reimbursement for reasonable travel, accommodation and food expenses.


Please click below for the information sheet:

Audentes Seroprevalance Information Sheet



If you would like to get in touch with the Newcastle team the contact details as follows:

Ruth Wake

Neuromuscular Research Nurse

The John Walton Muscular Dystrophy Research Centre

International Centre for Life

Newcastle upon Tyne

NE1 3BZ

T 0191 241 8865

ruth.wake4@nhs.net