Pompe Webinars from AMDA

Pompe Webinars from AMDA

Posted On: 21/03/2023

The USA patient organisation AMDA hold very interesting webinars throughout the year. The next one concerns two important topics.

Link to the Webinar description and registration page
Date: Tuesday, March 28, 2023
Time: 7pm BST

Patient Reported Outcomes (PROs)
PROs are important measures of the benefits that patients gain from treatments. However clinical trials don’t always use measures that are very meaningful to patients, for example being able to walk outdoors without tripping may be more important to you than adding 2 metres in a 6-minute walk Test (6MWT). Nadine van der Beek will discuss how the IPA/Erasmus survey has helped to determine the most appropriate PROs for Pompe.

IPA/Erasmus Survey
There is soon to be a new release of the survey that has been used since 2004 to describe the natural history of Pompe disease, monitor treatment benefit, and develop PROs. We hope to attract many more people to sign up to the survey as numbers have dwindled over the years. Maudy Theunisson will present results from past surveys, introduce the most recent changes and give a glimpse into the future for this important survey.