Jan 6, 2015 | NEWS
BioMarin are pausing further recruitment for their BMN-701 clinical trial and have provided this programme update: January 2015 BioMarin is pleased to update the Pompe patient community on the progress made in our programme to develop a treatment for people living...
Dec 15, 2014 | NEWS
The fifth annual AMDA Research Grant (the 2014 Helen Walker Research Grant) was awarded to Dr. Giancarlo Parenti with the Federico II University in Naples, Italy for his Project: “Analysis of Circulating and Tissue Specific microRNAs in Pompe Disease.” Despite...
Oct 17, 2014 | NEWS
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®...
Aug 1, 2014 | NEWS
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...
Jul 13, 2014 | NEWS
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,...
May 27, 2014 | NEWS
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...
May 4, 2014 | NEWS
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...
Feb 23, 2014 | NEWS
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...
Feb 11, 2014 | NEWS
To read the Program Update, please click here.
Jan 1, 2014 | NEWS
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...
