Welcome to the International Pompe Association (IPA)
Press Release: For Immediate Release
December 16, 2014
As many of you are probably aware, on December 13, 2014 an interview was published in Argos (a Dutch radio program) regarding a potential new production method for a treatment for Pompe disease. A short summary of the interview was made available in English on the website: www.utrechtcentral.com.
The International Pompe Association (IPA) strongly supports any research into Pompe disease that will increase disease knowledge, improve current treatments (through better efficacy or better price), or lead to better disease management. We also strongly believe in providing the Pompe community with clear, unbiased information regarding developments in the field of Pompe research.
After the publication in Argos, the IPA and our member organizations have received numerous inquiries regarding the announcement in the article that “a new, more economical method” for producing enzyme replacement therapy for Pompe disease could be possible within the next two years.
The IPA fully supports any efforts that will lead to more cost-effective treatments, as well as more effective treatments in general. However, at this time it is also important to understand the context in which the statements in the article were made.
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.