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About Pompe Disease
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Patient-Focused Publications
Patients Helping Patients
Research Papers
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About the IPA
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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...
Author:
IPA
News
July 13, 2024
Amicus Therapeutics Announces Approval and Launch of New Pompe Disease Therapy in the European Union
Amicus Therapeutics announces that the European Commission (EC) has granted approval for Opfolda® (miglustat), an enzyme stabilizer of cipaglucosidase...
Author:
IPA
News
June 28, 2023
Patient Registries for Pompe Disease Survey
The IPA invites you to share your perspective about Patient Registries for Pompe disease.
Author:
IPA
News
June 4, 2023
2023: Every Move Counts
The IPA is excited to announce its plans for International Pompe Day. This year, we are focusing on...
Author:
IPA
International Pompe Day
May 17, 2023
