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Rabbit Milk, Hamster Cells & Singing Quails
Developing the first drug for Pompe disease. First published on October 28, 2024 by Maryze Schoneveld van der Linde and...
Author:
IPA
News
July 8, 2026
First dosing in AskBio’s LOPD gene therapy trial
AskBio has dosed the first participant in an AAV-based gene therapy approach for adults (Phase 1/2 PROGRESS-GT LOPD).
Author:
IPA
News
May 13, 2026
First patients enrolled in Shionigi’s new Pompe disease study
Patients are now being enrolled in this clinical trial for a new substrate reduction therapy for Pompe disease.
Author:
IPA
News
April 25, 2026
Survey: Growing global participation and next steps
The long-running international Pompe Survey by IPA and Erasmus MC, Rotterdam, continues to expand its reach and impact. Established in...
Author:
IPA
News
March 20, 2026
BioMarin acquires Amicus Therapeutics
BioMarin Pharmaceutical expands its portfolio with the acquisition of Amicus Therapeutics, with closure in Q2 of 2026.
Author:
IPA
News
December 19, 2025
Formulation Survey
This research is to understand your experience with Pompe disease and the unmet needs that currently exist with treatment today.
Author:
IPA
News
July 25, 2025
Dare to Dream
Professor Ans van der Ploeg, MD, PhD, is retiring.
Author:
Annic Kolbrück
News
July 1, 2025
In Memoriam: Tiffany House
Tiffany was a force. A driving energy connecting patients, scientists, clinicians, and industry—always pushing for better care, stronger research, and...
Author:
IPA
News
June 9, 2025
In Memory of Tiffany House
It is with hearts filled with grief that we share with you Tiffany House has passed away, May 26, 2025.
Author:
IPA
News
May 26, 2025
In Memory of Maryze Schoneveld van der Linde
Maryze passed away peacefully on Wednesday, May 14. Maryze worked tirelessly for patients around the world.
Author:
Tiffany House
News
May 18, 2025
International Pompe Day: 15 April 2025 – Run, Walk or Roll
Run, Walk or Roll for Pompe disease on International Pompe Day, April 15, 2025!
Author:
IPA
Events
April 1, 2025
Spark Therapeutics Stops Its Pompe Program
The IPA has been informed that Spark Therapeutics has realigned its portfolio and made the difficult decision to stop its...
Author:
IPA
News
July 13, 2024
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