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...

The AMDA is excited to announce a research grant opportunity for applicants who are involved in Pompe DiseaseA rare genetic disease in which the body cannot properly break down glycogen, leading to buildup tha... More 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...

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. More. Hear the latest advancements, including newly approved...

Project title: AutophagyThe cell's internal cleanup system that removes damaged or unneeded material. More control of glycogenA stored form of sugar used for energy. More in skeletal muscleMuscles that control movement and are commonly affected in Pompe disease. More 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...

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...

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...

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...

The AMDA is excited to announce a research grant opportunity for applicants who are involved in Pompe DiseaseA rare genetic disease in which the body cannot properly break down glycogen, leading to buildup tha... More 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...

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...

At International Pompe Day the International Pompe Association kicked off the Every Move Counts project. This project focuses on raising awareness of the importance of movement. We all have different abilities and levels of disability, but the important thing to...