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 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...
Nov 7, 2023 | PAST WEBINARS
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...
Oct 17, 2023 | PAST WEBINARS
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...
Oct 10, 2023 | PAST WEBINARS
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...
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...
Sep 27, 2023 | PAST WEBINARS
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...
Aug 10, 2023 | PAST WEBINARS
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...
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...
