There are currently three enzyme replacement therapies (ERT) commercially available for Pompe disease in the UK (two approved for Infantile patients).
Patients, caregivers and clinicians have identified a need for support materials to help patients make informed decisions about their treatment choices and the management and monitoring of their condition at their specialist centre.
PSN, along with clinicians, have created a treatment guide for patients to provide information about the three therapies and other treatment or management options available. The guidelines are designed to empower and inform people with Pompe disease on their treatment journey and help them co-ordinate with their medical team in shared decision-making.
We thank industry partners, Sanofi UK and Amicus Therapeutics for providing funding to develop and print this guide.
Download the Guidelines:
We also created appointment sheets to help patients and caregivers to better prepare for their appointments. They can be downloaded and printed or filled out as a pdf.
Download the Appointment Sheets:
Additional up-to-date information about the available treatment options and research about their effectiveness is provided in this document which is designed to be an "insert" for the printed Guideline Booklets.
Download the insert here:
Pompe Support Network is happy to provide printed copies for anyone who does not have a printer, or for organisations who would like to supply the guidelines for patients or caregivers. Contact us at hello@pompe.uk to request them.