Welcome to the International Pompe Association (IPA)
The first meeting of the IPA Community Advisory Board (CAB) was recently held in San Antonio Texas on October 28, 2019.
The IPA established its Community Advisory Board (CAB) for Pompe disease in 2019 and consists of 24 individuals from the Pompe patient community, including patients, family members of patients, and patient organization leaders from different countries and continents around the world, representing the range of varieties of Pompe disease, from infantile to late-onset Pompe disease. During the meeting, the CAB members offered their experience and expertise to sponsors of clinical research, on overall program development, single clinical trials, hopes for future therapies, and other aspects beyond the research program, with the ultimate goal of ensuring that clinical studies are designed to consider the real needs of patients.
The meeting was moderated by James Valentine, an independent third-party moderator, and was extremely productive, setting a high standard for future CAB meetings.
- Summary of Key-Takeaways (PDF, 197 KB)
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.
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: