Welcome to the International Pompe Association (IPA)
The SSIEM (Society for the Study of Inborn Errors of Metabolism) Annual Symposium was held in Rotterdam, the Netherlands, from September 3 to September 6, 2019. This year’s Symposium President was Dr. Ans van der Ploeg.
The symposium abstracts are published on the Journal of Inherited Metabolic Disease, JIMD, and can be found at this link.
Here is a list of the relevant titles for Pompe disease that can be found in the journal at the above link:
November 3, 2019, The Straits Times
Rare Disease Fund now covers Pompe disease, a rare inherited neuromuscular disorder
Good news for our friends in Singapore: The Straits Times reports that "citizens can now apply for financial aid to help with their medical expenses for the disease". This is the fourth rare disease covered in Singapore - one of the other diseases is Gaucher disease, like Pompe a lysosomal storage disease.
The IPA has authored an article titled “The role of patient advocacy organizations in shaping medical research: the Pompe model,” and we are excited to announce that it has been accepted for publication.
The article focuses on the Pompe Model, a term describing the long-standing relationship between the patient community, the medical/scientific community, and industry. Over the last 20 years, we have seen that as a united community of patients, family, friends, physicians, scientists, and industry, working together, we have an undeniable strength and we have made, and will continue to make, great strides in learning more about Pompe Disease and in working towards treatments and disease management.
The article is published in the Annals of Translational Medicine, an international, peer-reviewed journal, and can be found here.