Welcome to the International Pompe Association (IPA)
Pompe News
The American Society of Gene & Cell Therapy, ASGCT, has recently developed a series of user-friendly resources to educate on gene therapy for Pompe disease, including a short animated video and infographic.
Other resources (e.g., Gene Therapy Basics, Different Gene Therapy Approaches, Gene Therapy Mythbusting) are also available on the ASGCT website.
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.