“On June 26, 2022, Astellas Pharma, Inc. (“Astellas”) issued a press release announcing that the U.S. Food and Drug Administration
The AMDA is excited to report that the final Voice of the Patient Report for the Pompe Patient-Focused Drug Development
Aro Biotherapeutics to Present New Preclinical Data Highlighting the Potential of Centyrin-siRNA Conjugates for the Treatment of Pompe DiseaseA rare genetic disease in which the body cannot properly break down glycogen, leading to buildup tha... More. To
April 11, 2022
AMDA Zoom Webinar With Sanofi
Webinar Details Title: POMPE REGISTRY: 18 YEARS OF LEADERSHIP AND CONTRIBUTIONS Date: Monday, April 11, 2022 Speaker:Joseph Bender, MD, MBA,
February 28, 2022
2022 PCMA Pull for Pompe Fundraiser
The AMDA is excited to announce that the 11th Annual PCMA’s Pull for Pompe will take place on Saturday, April
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
October 14, 2021
AMDA and Spark Webinar: Understanding Gene Therapy Research and the Potential Applications for Late Onset Pompe Disease
To watch the Webinar, please click here!
September 15, 2021
Re-Scheduled–Friday, Oct 8, 2021: AMDA and Spark Webinar: Understanding Gene Therapy Research and the Potential Applications for Late Onset Pompe Disease
Hi All, We are excited to announce that the webinar is on for Friday, October, 8, 2021 at 1 PM
PARIS – August 6, 2021 – The U.S. Food and Drug Administration (FDA) has approved Nexviazyme® (avalglucosidase alfa-ngpt) for the
Amicus’ AT-GAA Shows Clinically Meaningful & Significant Improvements in Both Musculoskeletal and Respiratory Measures in Late-Onset Pompe DiseaseA form of Pompe disease that begins after infancy and usually progresses more slowly. More Compared to