A letter from Sanofi to the global Pompe community, regarding the disruption of supplies of their treatments for...

Supply constraints affecting Sanofi's Pompe disease treatments are expected to continue to affect patients in different parts of...

Developing the first drug for Pompe disease. First published on October 28, 2024 by Maryze Schoneveld van der...

AskBio has dosed the first participant in an AAV-based gene therapy approach for adults (Phase 1/2 PROGRESS-GT LOPD).

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...