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.

The IPA is excited to announce its plans for International Pompe Day. This year, we are focusing on raising awareness of the importance of movement. We all have different abilities and levels of disability, but the important thing to remember and focus on is that “Every Move Counts.” We should celebrate what we can do, not focus on what we can’t.

But we need your help! We want to show all the ways that individuals in the Community show that Every Move Counts. Do you have modified physical therapy that you can share? Do you keep moving by playing with your dog? Do you focus on music or other hobbies to keep yourself moving? The ways that we can show that Every Move Counts will be as diverse as our community! By sharing how YOU believe Every Move Counts, you can help others in our community! If you are interested in being part of this project, please reach out to us at info@worldpompe.org. Our plan is to collect these stories, videos, pictures, etc. and will then work with experts in the field to compile a set of resources that will be available on the IPA website. We will need representatives from all around the world, and across the disease spectrum so please consider joining our Project!

Every Move Counts also relates to developments in Pompe treatment and management, and our daily lives. Therefore, our SECOND project for International Pompe Day is that we are requesting submissions for a 2024 Calendar that we will create. We would like to ask our community to submit artwork for a calendar that we will distribute to our members and to others in the community to remind us throughout next year to not give up—Every Move Counts! In research, in drug development, and in our lives. So please submit artwork to info@worldpompe.org that shows what Every Move Counts means to you!

Finally, the IPA created an awareness poster (4 versions) that is available on our website: PDF 4 posters, JPG poster 1, JPG poster 2, JPG poster 3, JPG poster 4. Please feel free to download it and share it as much as you like to help raise awareness of Pompe. Perhaps at you physical therapy center, or gym, or school, or mosque or church. Wherever you want to share it, we encourage you to share it!

Happy International Pompe Day!
#TOGETHERWEARESTRONG

Over the last 2 years, it is clear that the COVID-19 pandemic has had wide reaching effects on people all over the world. To better understand the impact of the Covid 19 Pandemic on the worldwide Pompe community, the International Pompe Association and the Erasmus MC are undertaking a one-time international survey of Pompe patients. The purpose of this survey is to look at both the effects of the pandemic on the life-style of Pompe patients, as well as the effects of infections with COVID-19. We believe that it is critical to gather this data so that we can properly and timely document these effects and we would like to ask your help.

To complete the survey would you please contact your local Pompe organisation who will provide you with a link to the survey. To make this as easy as possible, versions of the survey are available in a number of languages.

All Pompe patients over the age of 16 years old are welcome to complete the survey.

All information will be deidentified and the survey has been approved by the Erasmus MC ethics committee. Please see the Erasmus MC Patient Privacy Information https://www.erasmusmc.nl/en/patient-care/privacy

If there is not an IPA affiliate in your country or region, or you need assistance, please email info@worldpompe.org   

Thank you for your help,

Tiffany House

Chair, International Pompe Association.