Aug 1, 2024 | ARCHIVES, NEWS
Project title: Autophagy control of glycogen in skeletal muscle 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...
Aug 1, 2024 | ARCHIVES, NEWS, WEBINARS
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...
Jun 23, 2024 | ARCHIVES, NEWS
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...
Feb 10, 2024 | ARCHIVES, EDITORIALS
By: IPA posted on: May 27, 2019 The IPA has authored an article titled “The role of patient advocacy organizations in shaping medical research: the Pompe model,” and we are excited to announce that it has been accepted for publication. The article focuses on...
Jan 29, 2024 | NEWS, ARCHIVES
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...
Dec 28, 2023 | ARCHIVES, NEWS
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...
Oct 4, 2023 | ARCHIVES, NEWS
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...
Aug 3, 2023 | NEWS, ARCHIVES
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...
Aug 2, 2023 | NEWS, ARCHIVES
Dear Pompe Warriors and Patient Community, We are excited to announce the upcoming 2024 Acid Maltase Deficiency Associate – International Pompe Association (AMDA-IPA) Conference, a momentous event dedicated to advancing knowledge, support, and advocacy for...
Aug 2, 2023 | NEWS, ARCHIVES
Enter July 23rd – August 31st at RareArtist.org The Rare Artist Program was established in 2010 to exhibit the unique gifts of individuals impacted by rare disease to tell their story through art. Now in year 13, the Rare Artist Annual Contest is focused on...
