An Open Letter to the Pompe Community

On October 28, Amicus Therapeutics issued a press release with an update on the progress of our Pompe program as we received information from the US Food & Drug Administration (FDA) on the regulatory status of our next-generation therapy for the treatment of...

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International Survey on Pompe Disease and Covid-19

Over the last 2 years the COVID-19 pandemic has had wide reaching effects on people all over the world. To better understand the impact of the Covid 19 Pandemic on the worldwide Pompe community, the International Pompe Association and the Erasmus MC are undertaking a...

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2022 PCMA Pull for Pompe Fundraiser

The AMDA is excited to announce that the 11th Annual PCMA’s Pull for Pompe will take place on Saturday, April 30, 2022 at the National Shooting Complex in San Antonio, Texas! The Pull for Pompe clay shooting event is designed for all ages and ability levels. Please...

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10th Annual PCMA Pull for Pompe!

The AMDA is excited to announce that the 10th Annual PCMA’s Pull for Pompe will take place on Saturday, April 4, 2020 at the National Shooting Complex in San Antonio, Texas! The Pull for Pompe clay shooting event is designed for all ages and ability levels. Please...

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Audentes Pompe Program Update

Audentes has released a Pompe Program Update. It includes information regarding FORTIS, their clinical trial for gene therapy for Pompe. To read the Pompe Program Update, please click here.

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Patient Focused Drug Development Meeting—UPDATE

As everyone knows, due to the ongoing COVID-19 pandemic, the Patient-Focused Drug Development Meeting (PFDD) that was originally scheduled for March 09, 2020, was postponed. It has been moved to a Virtual Meeting that will take place Monday, July 13, 2020, from...

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2020 PCMA Pull For Pompe!—Cancelled

Update: July 8, 2020 It is with great sadness and disappointment that we hereby announce the cancellation of the 2020 Pull For Pompe Shoot due to the on-going COVID-19 pandemic. When we initially postponed the Shoot to August 15, we had high hopes that the virus would...

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