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 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...

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...

In 2014, the Pompe Registry recognized and celebrated an important milestone: the 10-year anniversary of the Registry. Started in 2004 and sponsored by Genzyme, a Sanofi company, the Pompe Registry was established as a global, voluntary, observational database to...

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 Sanofi Genzyme today presented data from NEO1, its Phase 1/2 clinical study evaluating the investigational novel enzymeA protein that helps the body carry out chemical reactions....

The AMDA is excited to announce that “Inspired by Pompe—High Protein, Reduced Carb Cooking” Cookbooks are availble for sale. This cookbook was written by fellow Pompe patient Sharon Krueger, and all proceeds will go towards the AMDA. Click here to view the Preface and...

Reveglucosidase Alfa Trends Positive in Respiratory Muscle Strength and Endurance Endpoints in Patients Previously Treated With Enzyme Replacement Therapy SAN RAFAEL, Calif., Jan. 11, 2016 (GLOBE NEWSWIRE)—BioMarin Pharmaceutical Inc. (Nasdaq:BMRN) today announced...

The AMDA is excited to announce that the 2016 PCMA of Texas Pull for Pompe will take place on Saturday, April 16 at the National Shooting Complex in San Antonio, Texas! This is the 6th Annual Pull for Pompe hosted by the PCMA of Texas. Since it’s launch in 2011, over...

CRANBURY, N.J., Dec. 22, 2015 (GLOBE NEWSWIRE)—Amicus Therapeutics (Nasdaq:FOLD), a biopharmaceutical company at the forefront of rare and orphan diseases, today announced that its investigational new drug (IND) application, submitted to U.S. Food and Drug...

The sixth annual AMDA Research Grant (the 2015 Helen Walker Research Grant) was awarded to Dr. Rosa Puertollano and Dr. Nina Raben for their Project: “CurcuminA natural compound being studied for its potential role in supporting cellular processes.: Can it promote cellular clearanceThe process by which cells remove waste and damaged materials. in 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.?.” The aim of this project is to test the possibility of...

The AMDA is excited to announce a research grant opportunity for applicants who are involved in Pompe Disease research. This grant opportunity was made possible by a fundraiser hosted by the Precast Manufacturer’s Association of Texas (PCMA of Texas) on April 18,...