2019 AMDA/IPA International Conference

The AMDA/IPA International Pompe Patient and Scientific Conference will take place in San Antonio, Texas from October 25-27, 2019. Please click here for the 2019 Conference Registration Form.  Each family, speaker, patient organization representative, industry...

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2019 PCMA’s Pull For Pompe!

The AMDA is excited to announce that the 2019 PCMA’s Pull for Pompe will take place on Saturday, April 13th at the National Shooting Complex in San Antonio, Texas! This is the 9th Annual Pull for Pompe hosted by the PCMA. The Pull for Pompe clay shooting event is...

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AMDA Webinar: Spark Therapeutics Gene Therapy Research

SPARK THERAPEUTICS GENE THERAPY RESEARCH WEBINAR Please join the AMDA for a webinar focused on Spark Therapeutics and gene therapy research Take this opportunity to learn more about Spark as a company and the field of gene therapy research. We will be joined by...

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In Memory of Henri Termeer

he AMDA was saddened to learn of the sudden passing of Henri Termeer, former CEO of Genzyme. We have known and worked with Henri since the late-90s through his role as the CEO of Genzyme. We will never forget how he always took the time to talk to patients and their...

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Pompe in the News: What Happened to the Truth?

On February 28, 2017—Rare Disease Day—Pompe disease was once again the subject of national media attention. During his Joint Address to Congress, President Trump brought national attention to patients suffering from rare diseases in general, and Pompe disease in...

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2017 PCMA of Texas Pull for Pompe!

The AMDA is excited to announce that the 2017 PCMA of Texas Pull for Pompe will take place on Saturday, March 25th at the National Shooting Complex in San Antonio, Texas! This is the 7th Annual Pull for Pompe hosted by the PCMA of Texas. The Pull for Pompe clay...

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2016 Helen Walker Research Grant Awarded

The seventh annual AMDA Research Grant (the 2016 Helen Walker Research Grant) was awarded to Dr. Frédéric Lofaso with the Hôpital Raymond Poincaré in France for his Project: “Acid Maltase Deficiency Myopathy Follow-Up.” The aim of this project is to improve the...

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BioMarin Update for the Pompe Community Regarding BMN 701

The IPA Board was provided with the following statement regarding Biomarin’s BMN-701 study. If you have any questions or concerns please contact the IPA at . If and when more information is available the IPA will inform you as soon as possible. BioMarin greatly...

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