Patient Registries for Pompe Disease

Patient Registries for Pompe Disease

Posted On: 15/06/2023

The International Pompe Association would like to request your assistance in conducting a brief (only a few questions) survey on the topic of Patient Registries for Pompe Disease.

This should not take more than 10 minutes of your time, and will benefit us greatly in on-going discussions with industry, medical and scientific experts in Pompe, and third parties.

Before we begin, we would like to briefly define what a Patient Registry consists of when developed for research or regulatory purposes:

A formal Patient Registry includes clinical (and sometimes patient reported outcome) information, which would be updated by your treating specialist at your or your child’s clinical appointment. It is an organized system for the collection, storage, retrieval, analysis, and dissemination of information. The purpose of collecting this data is to allow for greater research into Pompe in terms of how patients do over time and over different disease management strategies.

Many of you may already be enrolled in the Sanofi Patient Registry, as well as participate in the IPA/ Erasmus MC Pompe Survey, and local registries maintained by your treating physician. Whether or not you currently participate in a Registry or Survey, we are interested in your thoughts.

Now available in 8 languages until June 30, 2023.


Thank you for your time.