Welcome to the International Pompe Association (IPA)
Youtube video by Genzyme Corp., published April 08, 2015
(Original video size: 854x480px)
In 2014, the Pompe Registry recognized and celebrated an important milestone: the 10-year anniversary of the Registry. Started in 2004 and sponsored by Genzyme, a Sanofi company, the Pompe Registry was established as a global, voluntary, observational database to collect and report on the natural history and outcomes of patients with Pompe disease through routine clinical practice, regardless of patient treatment status. This video highlights the importance of the Registry through interviews with physicians in the field and patients with Pompe disease. (Source: https://www.youtube.com/watch?v=hnjzV7dkbhs)
The video includes statements of Pompe medical professionals and Pompe patients participating in the registry:
- Dr. Mark Roberts, UK
- Dr. Sharon Krueger, First Pompe Registry Patient
- Dr. Priya Kishnani, USA
- Dr. Mary-Alice Abbott, USA
- Dr. Edward Cupler, Saudi Arabia
- Dr. David Stockton, USA
- Prof. Antonio Toscano, Italy
- Prof. Juan Llerena, Brazil
- Abby Phelps, Pompe Registry Patient
- Prof. Ans van der Ploeg, The Netherlands
- Dr. Barry Byrne, USA
- Dr. Yin-Hsiu Chien, Taiwan
- Dr. Kenneth Berger, USA
- Prof. Wolfgang Müller-Felber, Germany
- Dr. Paula Clemens, USA
- Dr. Virginia Kimonis, USA
- Dr. Laura Case, USA
The Pompe Registry is an important program that Genzyme sponsors and administers. A disease registry contains medical information on patients with a specific condition, in a database that can be analyzed and used by physicians treating patients with the same condition. Registries have proven to be especially valuable in rare diseases like Pompe disease. Information submitted to the Pompe Registry will be maintained as confidential in accordance with applicable national privacy regulations and other state and local laws related to medical information. (Source: https://pompe.com/patients/pompe-registry.aspx)
- Information for patients on the Registry website: https://www.registrynxt.com/Home/For-Patients-and-Families
- Information about the objectives and other aspects of the Registry (for Healthcare Professionals): https://www.pompe.com/healthcare-professionals/pompe-registry.aspx
- Registration for Healthcare Professionals on the Registry website: https://www.registrynxt.com/Public-Registration
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.
Anybody aware of the upcoming Pompe conference 30 October - 1 November in San Antonio TX, USA? More information and agenda here (click link to open).
Become part of the conference by following the Twitter hashtag #AMDAIPA15 - your thoughts and tweets are welcome. But even if you have no Twitter account you can read the tweets here: https://twitter.com/hashtag/AMDAIPA15.
After years of hard work, persistence, and commitment, the Australian Pompe Association (APA) has achieved its goal--adult Pompe patients will finally be able to access treatment for Pompe disease beginning September 1, 2015.
Myozyme, a very expensive therapy, is the only treatment available for this life-threatening rare disease. On July 27, 2015, Sussan Ley, the Australian Minister for Health, approved Myozyme to be added to the Life Saving Drug Programme.
Pompe disease is a spectrum disease that can affect infants, children, and adults. Severity of the disease is usually determined by age of onset, but it can be fatal at any stage.
Myozyme has been available in many countries since 2006 after receiving approval by the regulatory authorities both in Europe (EMEA) and in the USA (FDA). It is the only approved therapy for Pompe disease.
In Australia, infantile-onset patients have been able to access treatment through the Life Saving Drug Programme since 2010; childhood onset patients, ages 2-18, have had access to treatment since February 2015. Now, adult Pompe patients will also be eligible for treatment starting in September 2015. Without Government subsidy through the Life Saving Drug Programme, Myozyme would be cost prohibitive to most Australians.
Helen Walker, a founding member of the APA and a board member of the International Pompe Association (IPA), initiated the "fight" to have treatment available for all Pompe patients in Australia. She was dedicated to this endeavour, but did not live to see the “fruits of her labor.” She died in 2012.
Continuing her legacy is the current president of the APA, Raymond Saich. He and other dedicated members of the APA continued to push this initiative forward, and they have finally managed to make it a reality. Now all Australian Pompe patients can receive this treatment as do patients in other countries throughout the world.
The IPA applauds the dedication and persistence of the APA. The determination of the Australian Pompe Association (APA) exemplifies the slogan of the internation Pompe community represented by the IPA: “Together We Are Strong.”
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