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 Disease.”  Respiratory insufficiency is a serious, and...

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,...

To read press release, please click here.

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...

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...

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...

San Rafael, Calif, March 19, 2013 – BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) announced today results from POM-001, the Phase 1/2 trial for BMN-701, a fusion protein of insulin-like growth factor 2 and acid alpha-glucosidase (IGF2-GAA) for the treatment of...

The PCMA of Texas invites you to enjoy a day of family fun benefitting Acid Maltase Deficiency, also known as Pompe Disease.  This event is designed for all ages and ability levels. It will be held at the National Shooting Complex in San Antonio, Texas. Please click...

The AMDA is excited to announce that the 2012 AMDA Research Grant Recipient is Dr. Nuno Raimundo of the University of Goettingen in Germany for his Project: “Mitochondria and Autophagy as Therapeutic Targets for Acid Maltase Deficiency.” Lysosomes are responsible for...

CRANBURY, NJ, January 4, 2013 – Amicus Therapeutics today announced positive preliminary results from all 4 dose cohorts in a Phase 2 study (Study 010) to evaluate the safety and pharmacokinetic (PK) effects of the pharmacological chaperone AT2220 (duvoglustat HCl)...

AT2220 Co-Administered with Enzyme Replacement Therapy Second Phase 2 Pharmacological Chaperone-ERT Co-Administration Study CRANBURY, NJ, US, December 1, 2011 – Amicus Therapeutics, a biopharmaceutical company at the forefront of developing therapies for rare...

On the 30th November 2012, Helen Walker OAM (Member of the Order of Australia) passed away. Helen was a board member of the International Pompe Association (IPA) and President of the Australian Pompe’s Association. Helen passed away at the age of 72 after having...

The path to diagnosis can be long and complicated and patients are frequently misdiagnosed along the way. In an effort to improve diagnostic accuracy, the AMDA and Genzyme collaborated on a questionnaire project in the first-quarter of 2012. The goal of the...

On Thursday, August 30, 2012 Dr. Arnold Reuser was decorated as a Knight in the Order of the Netherlands Lion by decree of Queen Beatrix of the Netherlands. The decoration was presented at the conclusion of the “Passion for Pompe” Program at the Arminius Church in...

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) aimed at...

I am very excited to announce that the 2012 PCMA’s Pull for Pompe was a HUGE success.  The PCMA of Texas (www.pcmatexas.org) raised almost $90,000.00 for Pompe Disease Research through its annual skeet-shooting fundraiser! Please click here for PCMA of Texas’s...

May 13, 2012—Program Update from BioMarin. For more information about the BioMarin trials please contact Sean McCarthy of BioMarin at: smccarthy@bmrn.com. The AMDA is also available to help answer any questions or concerns.  Please contact Tiffany House or Marsha...