We would like to invite all Pompe patients, or parents of Pompe children, to share your perspective about Patient Registries for Pompe disease. This brief survey 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.
If you’re able to complete this in English, please do so. We will follow it up soon in other languages, but we would like to get a snapshot now that will guide us in early discussions.
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 by Friday, May 19, 2023 .
www.surveymonkey.com/r/pompe-registries-230508
Background
A Patient Registry could be 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.