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 Disease Compared to
February 2, 2021
Spark Therapeutics Announces First Participant Dosed in Phase 1/2 Study of Investigational Gene Therapy for Late-Onset Pompe Disease
First participant dosed in the RESOLUTESM trial, a Phase 1/2 dose-escalation study of SPK-3006 Enrollment of approximately 20 total study
January 22, 2021
10th Annual PCMA Pull for Pompe!
The AMDA is excited to announce that the 10th Annual PCMA’s Pull for Pompe will take place on Saturday, April