The AMDA is excited to announce that the 2021 Helen Walker Grant for Pompe Disease was awarded to Dr. Peter Meinke and Dr. Benedikt Schoser for their Project entitled: "Generation of a platform for comparative testing of new treatments for people living with Pompe...

"On June 26, 2022, Astellas Pharma, Inc. ("Astellas") issued a press release announcing that the U.S. Food and Drug Administration (FDA) has placed a clinical hold on the FORTIS Phase 1/2 clinical trial evaluating AT845 ("the investigational gene therapy candidate")...

The AMDA is excited to report that the final Voice of the Patient Report for the Pompe Patient-Focused Drug Development meeting has been released. To view the report, please CLICK HERE!

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 that damages muscles and can affect breathing and, in some cases, the heart. More. To read the article, please click here.

The AMDA is excited to announce that the 11th Annual PCMA’s Pull for Pompe will take place on Saturday, April 30, 2022 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 cases, the heart. More research. This grant opportunity was made possible by the 2021 Pull for Pompe, a private fundraiser held by the Precast Manufacturer’s Association of Texas...

Hi All, We are excited to announce that the webinar is on for Friday, October, 8, 2021 at 1 PM CST. If you have already registered, you don't need to do anything. If you have NOT registered yet, it's not too late! Please click here to register. And, don't forget, we...

PARIS – August 6, 2021 - The U.S. Food and Drug Administration (FDA) has approved Nexviazyme® (avalglucosidase alfa-ngpt) for the treatment of patients one year of age and older with late-onset 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 cases, the heart. More, a progressive and debilitating muscle disorder that impairs...

Amicus’ AT-GAA Shows Clinically Meaningful & Significant Improvements in Both Musculoskeletal and Respiratory Measures in Late-Onset 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 cases, the heart. More Compared to Standard of Care in Pivotal Phase 3 PROPEL Study. Amicus Therapeutics, Inc. Download file     Download...

First participant dosed in the RESOLUTESM trial, a Phase 1/2 dose-escalation study of SPK-3006 Enrollment of approximately 20 total study participants is ongoing PHILADELPHIA, February 1, 2021 (GLOBE NEWSWIRE)— Spark Therapeutics, a member of the Roche Group (SIX: RO,...