Shionogi & Co., LTD., and Maze Therapeutics, Inc. have agreed to develop for market, MZE001, a new substrate reduction therapy drug that could be the first ever pill take orally to treat Pompe disease.

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OTHER NEWS

2024 PCMA Pull for Pompe Fundraiser

The AMDA is excited to announce that the 13th Annual PCMA’s Pull for Pompe will take place on Saturday, April 27, 2024 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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Rare Artist Contest is now Open for Submissions

Enter July 23rd - August 31st at RareArtist.org The Rare Artist Program was established in 2010 to exhibit the unique gifts of individuals impacted by rare disease to tell their story through art. Now in year 13, the Rare Artist Annual Contest is focused on providing...

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2023 PCMA Pull for Pompe Fundraiser

The AMDA is excited to announce that the 12th Annual PCMA’s Pull for Pompe will take place on Saturday, April 29, 2023 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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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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 Acid Maltase Deficiency Association

GET INVOLVED

Your generous contribution will go a long way into improving the quality of life of Pompe Disease Patients worldwide. Donations go towards Pompe Disease Research.

If you are interested in learning more about Pompe Disease and would like to make a contribution in support of necessary research, please contact us at:

info@amda-pompe.org

AMDA

PO Box 700248

San Antonio, Texas 78270 USA