Welcome to the International Pompe Association (IPA)
On the 17th - 19th October the Australian Pompe's Association held its first National patient and family support forum in Melbourne.The objective was to bring everyone together and provide advice on diet and exercise and provide some advice on living with Pompe disease.
With Australia being so vast, similar size to Europe, but with only 33 patients the task of bringing members together was going to be a challenge. Members are living in every state and territory and some members had flights of over 4 hours to join the meeting. Three of our New Zealand colleagues also made the journey across the Tasman to be at the meeting. The forum was important as many of the members being so isolated had never met another Pompe patient or one who is not related to them. The opportunity to learn from one another and to just see how others have conquested the challenge of getting up or negotiating a few steeps was so important.
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'!