January 23, 2019
2019 PCMA’s Pull For Pompe!
The AMDA is excited to announce that the 2019 PCMA’s Pull for Pompe will take place on Saturday, April 13th
December 20, 2018
Press Release: Amicus Therapeutics Announces First Patient Dosed in Phase 3 PROPEL Study
Amicus Therapeutics Announces First Patient Dosed in Phase 3 PROPEL Pivotal Study of AT-GAA in Patients with Pompe Disease Global
October 22, 2018
Spark Therapeutics Gene Therapy Research Webinar
Spark Therapeutics Gene Therapy Research Webinar Download an audio only version
October 8, 2018
Press Release: Spark Therapeutics Announces New Preclinical Data for Pompe Disease Gene Therapy
Spark Therapeutics Announces New Preclinical Data for Pompe Disease Gene Therapy Candidate Data from IND-enabling studies in three species support
September 25, 2018
Spark Therapeutics Gene Therapy Research Webinar
Webinar Details Title: Spark Therapeutics Gene Therapy Research Webinar Date: Tuesday, September 25, 2018 Speakers:Federico Mingozzi, PhD – Chief Scientific