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 that damages muscles and can affect breathing and, in some cases, the heart. Cambridge, Mass. – November 4,

The AMDA is excited to announce a research grant opportunity for applicants who are involved in Pompe Disease 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 the GAA enzyme., Genzyme

On the third annual International Pompe Day, the Pompe Community turned its attention to the importance of Early Diagnosis. For

In 2014, the Pompe Registry recognized and celebrated an important milestone: the 10-year anniversary of the Registry. Started in 2004

Results support beginning pivotal Phase 3 clinical trialA research study that tests new treatments or approaches in people. in Q2 2016 PARIS—(BUSINESS WIRE)—Sanofi and its specialty care global business unit