Welcome to the International Pompe Association (IPA)
The IPA is establishing a Community Advisory Board (CAB) for Pompe disease. A CAB is a group of volunteer patients/advocates who offer their expertise to public or private sponsors of clinical research on overall program development, single clinical trials and other aspects beyond the research program. A CAB advises several sponsors in the same fields and, by doing so, it ensures that research studies are designed to take into account the real needs of patients, resulting in higher quality research.
For this reason, the IPA is looking for, ideally, 15-20 CAB members, representing the range of varieties of Pompe disease (from infantile to late-onset Pompe disease), including parents and patients, spread around the world. Based upon the specific requirements/needs of the sponsor of particolar meetings, it is possible that the entire CAB may not be called upon every time.
- Must be over 18 years old;
- They should have the specific condition for which the CAB is being set up, be a parent of someone living with the disease, or be a member of an organisation dealing with the exact condition;
- They should have good English skills;
- They should be able to commit to a minimum of 2 face-to-face meetings a year (approx. 2 days each), and 2 other e-meetings;
- CAB members should adhere to the Code of Practices Guiding the Relations between Patients’ Organisations and the Healthcare Industry.
- They should be member of an organisation, not unaffiliated individuals.
Please write a letter stating why you wish to be a CAB member. Please include in the letter how you meet the above requirements and any experience you have that you think the Reviewers should be aware of. If you do not meet one of the requirements above, but believe you should be considered anyway, please explain why. Application letters should be sent to firstname.lastname@example.org.
The deadline to submit applications is October 31, 2018.
Personal data will be processed only for the purpose of this call for Applicants.
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.
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.
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:
- 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.
- 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.
- Submit both a question/thought and artwork that goes with it
Please submit your contribution by e-mail to email@example.com
The deadline for submissions is: Sunday 27th 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)