TOP NEWS
2025 PCMA Pull for Pompe Fundraiser
The AMDA is excited to announce that the 14th Annual PCMA's Pull for Pompe will take place on Saturday, April 26, 2025 at the National Shooting Complex in San Antonio, Texas! The Pull for Pompe clay shooting event is designed for all ages and ability levels. Please...
OTHER NEWS
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.
2013 PCMA of Texas’s Pull for Pompe a HUGE Success!
On April 20, 2013 the Precast Concrete Manufacturers’ Association of Texas (PCMA of Texas) hosted its Third Annual “Pull for Pompe” fundraiser. Two hundred (200) shooters and one hundred spectators (100) enjoyed a day of friendly competition and family fun at this...
Federal Advisory Committee Recommends Pompe Disease for Newborn Screening
WASHINGTON – May 17 2013 – In a long awaited meeting, the Secretary’s Discretionary Advisory Committee for Heritable Disorders in Newborns and Children (DACHDNC), in a vote of 11 – 2, recommended the addition of Pompe Disease to the recommended uniform newborn...
Callidus Biopharma Announces $ 4 .6 million in Series A Financing to Pursue Orphan Disease Therapies
DOYLESTOWN, PA (May 13, 2013)–Callidus Biopharma, Inc., a development-stage biotechnologycompany focused on creating breakthrough biologic drugs for a range of orphan diseases, announced today that it has closed on $4.6 million in Series A financing led by two...
Acid Maltase Deficiency Association
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