TOP NEWS
Texas Adds Pompe Disease to Newborn Screening Panel—A Historic Milestone Coinciding with the AMDA’s 30th Anniversary
Great news for the rare disease community—Texas has taken a big step by adding Pompe disease to its newborn screening panel! This major advancement in protecting the health of Texas infants also marks a meaningful moment for the Acid Maltase Deficiency Association...
OTHER NEWS
United States Pompe Community Update from Genzyme
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 alfa) manufactured at the 4000L scale and its impact on Myozyme®...
FDA and Genzyme Announce Label Expansion of Lumizyme in United States
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 enzyme replacement therapy for Pompe disease produced at the 4000L manufacturing...
2014 Helen Walker Research Grant Now Accepting Applications
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 5,...
BioMarin Doses First Patient in Phase 3 INSPIRE Trial with BMN 701 for the Treatment of Pompe
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 disease. BMN 701 is a novel fusion protein of insulin-like...
AMDA to Celebrate First International Pompe Day!!
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...
2014 AMDA Pull for Pompe
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...
Update on Genzyme’s neo-GAA Pompe Clinical Development Program
To read the Program Update, please click here.
2013 Helen Walker Research Grant Awarded
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 Disease.” Respiratory insufficiency is a serious, and...
2013 Helen Walker Research Grant Applications
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 20,...
Update on Genzyme’s neo-GAA Program
To read press release, please click here.
Acid Maltase Deficiency Association
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