TOP NEWS
A Heartfelt Thank You to the Krueger Family
The Acid Maltase Deficiency Association is deeply grateful to the Krueger family for their remarkable and generous donation to our organization. Their gift is a powerful testament to their compassion for all those affected by Pompe diseaseA rare genetic disease in which the body cannot properly break down glycogen, leading to buildup that damages muscles and can affect breathing and, in some cases, the heart. and their commitment to...
OTHER 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...
2024 PCMA’s Pull for Pompe Photo Gallery
On Saturday, April 27, 2024, the PCMA (Precast Concrete Manufacturers Association) held it’s annual Pull for Pompe fundraiser at the National Shooting Complex in San Antonio, TX. The Pull for Pompe clay shooting event was a day of family fun designed for all ages and...
2024 Helen Walker Research Grant For Pompe Disease 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 the 2024 Pull for Pompe, a private fundraiser held by the Precast Manufacturer’s Association of Texas...
Essential Update: New Treatment Approaches for Late-Onset Pompe Disease
The AMDA is partnering with Medlive to bring this exciting program to HCPs in the Pompe community. Join leading experts Dr. Tahseen Mozaffar and Dr. Barbara Burton as they discuss late-onset Pompe disease (LOPD)A form of Pompe disease that begins after infancy and usually progresses more slowly.. Hear the latest advancements, including newly approved...
2023 Helen Walker Grant Recipient
Project title: AutophagyThe cell's internal cleanup system that removes damaged or unneeded material. control of glycogenA stored form of sugar used for energy. in skeletal muscleMuscles that control movement and are commonly affected in Pompe disease. This year, the AMDA was fortunate to receive 13 grant applications focusing on both clinical research and basic science. It has been a challenge to select one. Dr. Wyant was chosen because he has embarked...
Join us for the first “Talking With Your Pompe Peeps” session on August 16, 2024!
We are excited to announce that the topic of our first "Talking With Your Pompe Peeps" session is "The Elementary School Experience: Building Success for IOPD/LOPD Children Through Collaboration and Partnership"! Join us August 16, 2024 at 1 PM CT for an open forum...
PEARL Trial-PrEnAtal enzyme Replacement for Lysosomal storage diseases
University of California - San Francisco (UCSF) is excited to announce the rebranding of their phase 1 clinical trail of In Utero Enzyme Replacement Therapy as the PEARL Trial-PrEnAtan enzyme Rreplacement for Llysosomal storage diseases. "We have rebranded our...
2024 PCMA Pull for Pompe Fundraiser
The AMDA is excited to announce that the 13th Annual PCMA’s Pull for Pompe will take place on Saturday, April 27, 2024 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...
2023 Helen Walker Research Grant For Pompe Disease 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 the 2023 Pull for Pompe, a private fundraiser held by the Precast Manufacturer’s Association of Texas...
Amicus Therapeutics Announces FDA Approval and Launch of New Treatment for Pompe Disease
PHILADELPHIA, Sept. 28, 2023 (GLOBE NEWSWIRE) -- Amicus Therapeutics (Nasdaq: FOLD) today announced that the U.S. Food and Drug Administration (FDA) has approved Pombiliti™ (cipaglucosidase alfa-atga) + Opfolda™ (miglustat) 65mg capsules. This two-component therapy is...


