TOP NEWS
2024 Helen Walker Research Grant Recipient: Dr. Jaymin Upadhyay, Boston Children’s Hospital, Harvard Medical School
The winner of the 2024 AMDA Helen Walker Research Grant: Dr. Jaymin Upadhyay, Boston Children’s Hospital, Harvard Medical School Project Title: Implementation of At-Home Functional BiomarkerA measurable sign in the body that helps track disease progression or treatment response. More Methods in Patients with Pompe DiseaseA rare genetic disease in which the body cannot properly break down glycogen, leading to buildup tha... More. For the second year in a row, the...
OTHER NEWS
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...
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...
Sanofi Genzyme Begins Pivotal Phase 3 Trial of NeoGAA Investigational Second-Generation Therapy
Sanofi Genzyme Begins Pivotal Phase 3 Trial of NeoGAA Investigational Second-Generation Therapy for Pompe DiseaseA rare genetic disease in which the body cannot properly break down glycogen, leading to buildup tha... More Cambridge, Mass. – November 4, 2016 - Sanofi Genzyme, the specialty care global business unit of Sanofi, announced today that the first patient has been...
2016 Helen Walker Research Grant For Pompe Disease Now Accepting Applications
The AMDA is excited to announce a research grant opportunity for applicants who are involved in Pompe DiseaseA rare genetic disease in which the body cannot properly break down glycogen, leading to buildup tha... More research. This grant opportunity was made possible by the 2016 Pull for Pompe, a private fundraiser held by the Precast Manufacturer’s Association of Texas...
The History of PCMA of Texas’s Pull for Pompe! Thank you!
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...
IPA Statement on Biomarin’s June 9th announcement it will seek to out-license BMN-701
Please visit the IPA website to view the statement: http://worldpompe.org/index.php/news/590-ipa-statement-on-biomarin-s-june-9th-announcement-it-will-seek-to-out-license-bmn-701
UPDATE: 2015 AMDA International Pompe Patient and Scientific Conference – Summary Report
UPDATE: DVD recordings of the Conference are available for purchase through the AMDA. The recordings include all presentations and Question and Answer Sessions from the Conference, and can be purchased for $60 (shipping within the US included). To order your DVD set,...
Celebrating the Pompe Community
In recognition of the third annual International Pompe Day, and the 10 Year Anniversary of the approval of MyozymeA form of enzyme replacement therapy used to treat Pompe disease by providing a lab-made version of ... More, Genzyme developed a video highlighting how far the Pompe community has come in the last 10 years, and our hopes for the future. Please find the video...
Press Release: International Pompe Day 2016
On the third annual International Pompe Day, the Pompe Community turned its attention to the importance of Early Diagnosis. For Immediate Release April 15, 2016 will be the Third Annual International Pompe Day. The International Pompe Association (IPA), with the...


