Excerpt . . . . CRANBURY, N.J., May 15, 2017 —Amicus Therapeutics today announced positive functional data from initial patients

The AMDA is excited to announce that the 2017 PCMA of Texas Pull for Pompe will take place on Saturday,

The seventh annual AMDA Research Grant (the 2016 Helen Walker Research Grant) was awarded to Dr. Frédéric Lofaso with the

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,

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

The IPA Board was provided with the following statement regarding Biomarin’s BMN-701 study. If you have any questions or concerns

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: DVD recordings of the Conference are available for purchase through the AMDA.  The recordings include all presentations and Question

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