Welcome to the International Pompe Association (IPA)
Formation of a European network to develop a European data sharing model and treatment guidelines for Pompe disease
October 6th, 1014 in Naarden, The Netherlands
This workshop was co-sponsored by the Dutch ZonMw, Genzyme Europe BV, the International Pompe Association, Amicus Therapeutics and BioMarin Europe Ltd.
Pompe disease is a rare inheritable muscle disorder for which since 2006 enzyme replacement therapy (ERT) is available. Studies in infants showing improved survival were key to the market approval of this drug. Since 2006 several studies in children and adults have also shown effects of this treatment. Harmonized criteria to start or stop ERT for the whole spectrum of patients with Pompe disease are needed.
This workshop brought together a panel of physicians and researchers from various European countries with clinical experience in treating and following larger groups of patients with Pompe disease, or doing research in this disorder, as well as Wilma Treur, patient representative from the IPA.
The Acid Maltase Deficiency Association (AMDA) in the USA, one of the members of the IPA, took the intiative to create an International Pompe Day Commemorative Book. This 70+ page Commemorative Book highlights the international activities that occured on the First Annual International Pompe Day at April 15, and will serve as a permanent reminder of how 'Together We Are Strong'!
Press Release - For Immediate Release
October 1, 2014
The International Pompe Association (IPA) is excited to announce that it has been selected as a recipient of Genzyme's 4th Annual PAL Awards program for its "Pompe M-Power" Pilot Project.
The Genzyme PAL Awards grants program was launched in 2011 to encourage bold new ideas and programs to support the LSD community worldwide. The PAL program funds innovative projects in disease awareness and education, community mobilization, non-profit development and good governance activities, patient care and support programs.
"The IPA's Pompe M-Power Pilot Project is the first step in launching the international 'Pompe M-Power' program, which will identify, educate, and mentor the future Pompe leaders in our Community. We appreciate Genzyme and the PAL Award Review Committee's recognition of this important project and look forward to beginning this work with our Pilot Program in the second to third quarter of 2015," says Tiffany House (IPA Chair).