Patients are now being enrolled in this clinical trial for a new substrate reduction therapy for Pompe disease.

The long-running international Pompe Survey by IPA and Erasmus MC, Rotterdam, continues to expand its reach and impact....

BioMarin Pharmaceutical expands its portfolio with the acquisition of Amicus Therapeutics, with closure in Q2 of 2026.

This research is to understand your experience with Pompe disease and the unmet needs that currently exist with...

Professor Ans van der Ploeg, MD, PhD, is retiring.

Tiffany was a force. A driving energy connecting patients, scientists, clinicians, and industry—always pushing for better care, stronger...

It is with hearts filled with grief that we share with you Tiffany House has passed away, May...

Maryze passed away peacefully on Wednesday, May 14. Maryze worked tirelessly for patients around the world.

Run, Walk or Roll for Pompe disease on International Pompe Day, April 15, 2025!

The IPA has been informed that Spark Therapeutics has realigned its portfolio and made the difficult decision to...