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 disease, a progressive and debilitating muscle disorder that impairs...

Pregnancy Outcomes in Late-Onset Pompe Disease Life Download file

Amicus’ AT-GAA Shows Clinically Meaningful & Significant Improvements in Both Musculoskeletal and Respiratory Measures in Late-Onset Pompe Disease 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,...

The AMDA is excited to announce that the 10th Annual PCMA’s Pull for Pompe will take place on Saturday, April 4, 2020 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...

Audentes has released a Pompe Program Update. It includes information regarding FORTIS, their clinical trial for gene therapy for Pompe. To read the Pompe Program Update, please click here.

Spark Therapeutics has developed a “Frequently Asked Questions” document regarding their RESOLUTE clinical trial. Please click here to read.

As everyone knows, due to the ongoing COVID-19 pandemic, the Patient-Focused Drug Development Meeting (PFDD) that was originally scheduled for March 09, 2020, was postponed. It has been moved to a Virtual Meeting that will take place Monday, July 13, 2020, from...

Update: July 8, 2020 It is with great sadness and disappointment that we hereby announce the cancellation of the 2020 Pull For Pompe Shoot due to the on-going COVID-19 pandemic. When we initially postponed the Shoot to August 15, we had high hopes that the virus would...

With all of the questions and uncertainty surrounding the ongoing COVID-19 pandemic, the AMDA worked with Dr. Priya Kishnani and her group at Duke University on the following Question and Answer Fact Sheet. We hope that the Pompe Community will find the information...