Genzyme would like to share with you an important update regarding the recent United States (U.S.) Food and Drug Administration’s (FDA) approval of an expanded label for Lumizyme® (alglucosidase alfaA form of enzyme replacement therapy used to treat Pompe disease by providing a lab-made version of ... More) manufactured at the 4000L scale and its impact on Myozyme®...

To the United States Pompe Community: On August 1, 2014 the US Food and Drug Administration (FDA) and Genzyme issued press releases regarding the expansion of the Lumizyme® label (the enzymeA protein that helps the body carry out chemical reactions. More replacement therapy for Pompe diseaseA rare genetic disease in which the body cannot properly break down glycogen, leading to buildup tha... More produced at the 4000L manufacturing...

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 a fundraiser hosted by the Precast Manufacturer’s Association of Texas (PCMA of Texas) on April 5,...

San Rafael, Calif., May 27, 2014 – BioMarin Pharmaceutical Inc. announced today that it has dosed the first patient with BMN 701 (GILT-tagged Recombinant Human GAA) in the Phase 3 INSPIRE trial for Pompe diseaseA rare genetic disease in which the body cannot properly break down glycogen, leading to buildup tha... More. BMN 701 is a novel fusion protein of insulin-like...

Update: May 4, 2014 THE AMDA TO CELEBRATE THE FIRST INTERNATIONAL POMPE DAY WITH COMMEMORATIVE BOOK April 15, 2014 marked the International Pompe Association’s (IPA) first annual International Pompe Day.  In honor of the First International Pompe Day, the Acid Maltase...

On Saturday April 5, 2014 the AMDA will host the Fourth Annual Pull for Pompe at the National Shooting Complex in San Antonio, Texas.  Over the last three years over $380,000.00 (USD) has been raised through this event and all proceeds go towards the AMDA Research...

To read the Program Update, please click here.

The fourth annual AMDA Research Grant (named the 2013 Helen Walker Research Grant) was awarded to Dr. Barbara Smith with the University of Florida for her Project: “[Diaphragmatic] Pacing in Subjects with Pompe DiseaseA rare genetic disease in which the body cannot properly break down glycogen, leading to buildup tha... More.”  Respiratory insufficiencyA condition in which the breathing muscles are too weak to support normal breathing needs. More is a serious, and...

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 a fundraiser hosted by the Precast Manufacturer’s Association of Texas (PCMA of Texas) on April 20,...

To read press release, please click here.