TOP NEWS
2025 New Year Letter From AMDA President – Tiffany House
As we enter a new year, the Acid Maltase Deficiency Association (AMDA) wants to thank you for your unwavering support and dedication. Together, we have navigated through challenges and made significant strides for the Pompe community. Your support fuels our commitment...
OTHER NEWS
BioMarin to Advance BMN-701 for Pompe Disease to Next Phase of Development
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...
2013 PCMA of Texas’s Pull for Pompe set for April 20, 2013!
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...
2012 AMDA Research Grant Awarded
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...
Amicus Announces Positive Results from Phase 2 Chaperone-ERT Co-Administration Study
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)...
Amicus Therapeutics Announces First Patient in Phase 2 Study for Pompe Disease
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...
Pompe Community Loses Valued Advocate
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...
News 2012 AMDA/Genzyme Patient Diagnosis Survey Results
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...
News Dr. Arnold Reuser Decorated by Queen of Netherlands
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...
2012 AMDA Research Grant
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...
2012 PCMA Pull for Pompe Fundraiser Results
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...


