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...
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...
PHILADELPHIA, June 27, 2023 (GLOBE NEWSWIRE) — Amicus Therapeutics (Nasdaq: FOLD), a patient-dedicated global biotechnology company focused on developing and commercializing novel medicines for rare diseases, today announced that the European Commission (EC) has...
Webinar Details Title: When A Hospital and A Patient Organization Work Together & The Value of Patient Reported Outcomes Date: Tuesday, March 28, 2023 Speakers:Maudy Theunissen, MDNadine van der Beek, MD, PhD Presentation 1:Title: When A Hospital and A Patient...
The AMDA is excited to announce that the 12th Annual PCMA’s Pull for Pompe will take place on Saturday, April 29, 2023 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 Disease research. This grant opportunity was made possible by the 2022 Pull for Pompe, a private fundraiser held by the Precast Manufacturer’s Association of...
Webinar Details Title: Insurance 101: Learning the Basics Date: Tuesday, December 13, 2022 Speaker:Amy DeStefanis, LCSW, CCM Webinar Overview:Insurance 101 will guide the participants through a discussion of the main types of health insurance coverage, types of health...
On October 28, Amicus Therapeutics issued a press release with an update on the progress of our Pompe program as we received information from the US Food & Drug Administration (FDA) on the regulatory status of our next-generation therapy for the treatment of...
Over the last 2 years the COVID-19 pandemic has had wide reaching effects on people all over the world. To better understand the impact of the Covid 19 Pandemic on the worldwide Pompe community, the International Pompe Association and the Erasmus MC are undertaking a...
Webinar Details Title: In Utero Enzyme Replacement Therapy for Lysosomal Storage Diseases Date: Friday, September 16, 2022 Speaker:Jeniffer L. Cohen, MD, Assistant Professor of Pediatrics at Duke University Webinar Overview:Dr. Cohen will provide an overview of the...
