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 that damages muscles and can affect breathing and, in some...
We are excited to announce that the 2024 AMDA/IPA International Pompe Patient and Scientific Conference will take place in San Antonio, Texas from May 3rd-5th, 2024. Registration for the Conference is now open! Please click here for the 2024 Conference In Person...
Webinar Details Title: Development of Gene Therapy for Pompe Disease: Recent History Date: Tuesday, November 7, 2023 Speaker:Dr. Dwight Koeberl Webinar Overview:Dr. Koeberl will discuss the rationale for gene therapy for Pompe disease. He will provide a summary of...
Webinar Details Title: Defining Central Nervous System Abnormalities in Infantile and Late-Onset Pompe Disease Patients Date: Tuesday, October 17, 2023 Speakers:Raquel van GoolJaymin Upadhyay Webinar Overview:In this presentation, we will provide a short description...
Webinar Details Title: Lentiviral gene therapy for Pompe disease Date: Tuesday, October 10, 2023 Speaker:Dr. Pim Pijnappel, Associate Professor in Cell and Gene Therapy at the Erasmus MC University Medical Center, Rotterdam, the Netherlands Webinar Overview:The...
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...
Webinar Details Title: Spark Therapeutics Webinar: Trail Expectations – What to Expect Date: Wednesday, September 27, 2023 Speaker:Jacose Bell Webinar Overview:This presentation, developed by Spark Therapeutics, takes audience members through the introductory...
Webinar Details Title: New insights into Pompe disease since the advent of NBS Date: Thursday, August 10, 2023 Speakers:Dr. Priya Kishnani, MDErin Huggins, MS, CGC Webinar Overview:An overview of the lessons learned from newborn screening for Pompe disease and its...
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...
