We would like to share the latest publication based on the data generated through the IPA/ Erasmus MC Pompe survey. 

Together with the International Pompe Association (IPA), the Erasmus University Medical Center (Erasmus MC) based in Rotterdam, Netherlands, has systematically collected data on patients with Pompe disease since 2002. Earlier findings from the survey include the demonstration of a positive effect of ERT on survival. Using data from this survey, it was investigated whether ERT reduces the risk that a patient will need a wheelchair or respiratory support. This is the first study to provide evidence that ERT with alglucosidase alfa reduces the risk that adult patients with Pompe disease will become wheelchair dependent (patients who received ERT had a 64% smaller probability of becoming wheelchair dependent than an untreated patient). Nevertheless, a proportion of treated patients still become wheelchair dependent at some point in their life. Hence, while ERT shows positive clinical effects in adult patients with Pompe disease, it has been concluded that there is still room for improvement. With regard to the risk for starting respiratory support, no differences could be detected. The IPA survey is an open cohort into which patients are continually included. It has been shown to be a good reflection of the clinical spectrum of adult patients with Pompe disease.

Read more

Enzyme Replacement Therapy Reduces The Risk For Wheelchair Dependency In Adult Pompe Patients (PDF, 609 KB)
© The Author(s). 2018 Open Access. This article is distributed under the terms of the Creative Commons Attribution 4.0 International License (http://creativecommons.org/licenses/by/4.0/), which permits unrestricted use, distribution, and reproduction in any medium, provided you give appropriate credit to the original author(s) and the source, provide a link to the Creative Commons license, and indicate if changes were made. The Creative Commons Public Domain Dedication waiver (http://creativecommons.org/publicdomain/zero/1.0/) applies to the data made available in this article, unless otherwise stated.

Disclaimer

The IPA does not endorse any of the products, medications, treatments or information reported herein. Articles on the IPA web pages are intended for informational purposes, only. We strongly advise that you discuss all medications, treatments, and/or products with your physician.

At the International Pompe Association’s Annual General Meeting in Nottingham, the IPA Board and Members who were present discussed how to best raise awareness of Pompe for the 2018 International Pompe Day (April 15, 2018).

After a lot of discussion, the group decided that we want to take the opportunity to raise awareness for not just a day, but for a whole year. Therefore, the idea of creating a calendar was born.

Today, on International Pompe Day we put out a call to the International Pompe Community to help the IPA to create a calendar for 2019 that will keep Pompe in the minds and hearts of all recipients throughout the year. It will be a monthly calendar, with a photo/artwork and a thought-provoking statement or question for each month.

How you can participate

There are three ways that you can participate:

1. Submit a question/thought to be considered for inclusion on one month of the calendar
   Is there a question or thought that is on your mind about Pompe? Something that you would like to share with the doctors, scientists, companies, and other patients? Something that you think should be on everyone’s mind? If so, please submit the question/thought and an explanation of why you think it is important.
2. Submit a photo or picture of artwork to be considered for inclusion in the calendar
   Please include a description of the artwork–what it means to you, and what you hope to portray to the international Pompe community through the art work.
3. Submit both a question/thought and artwork that goes with it

Please submit your contribution by e-mail to webmaster@worldpompe.org

The deadline for submissions is: Sunday 27^th May 2018.

We look forward to seeing everyone’s submissions and to creating a calendar that shows the world that “Together We Are Strong.”

Flyer: 2019 Pompe Calendar (PDF, 970 KB)

The IPA would like to thank everyone who participated in the Talent of Pompe (ToP) Awards Contest on the occasion of the Fifth Annual International Pompe Day!

The IPA is very excited to announce the winners in each category:

Age 0-7

First Place: Abdurrahman Amerat

The sun gives us light, energy and it grows food to make everyone strong. It also makes the universe powerful.