December 8, 2019
2019 AMDA/IPA Conference Report Available
On October 25-27, 2019, almost 200 Pompe patients and their families, scientists, doctors, and industry representatives from around the world
November 22, 2019
Patient Focused Drug Development Meeting to be held March 09, 2020
The AMDA is excited to announce that it is partnering with the MDA to host a Patient Focused Drug Development
October 1, 2019
The Common C.-32-13T G Splicing Variant of GAA Gene
Webinar Details Title: The common c.-32-13T>G splicingA normal step in how the body reads gene instructions. If splicing goes wrong, the wrong protein may be made. variant of GAA geneThe gene responsible for making the GAA enzyme.: from functional characterization to the identification of new therapeutic
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. research. This
August 17, 2019
2019 AMDA/IPA International Conference
The AMDA/IPA International Pompe Patient and Scientific Conference will take place in San Antonio, Texas from October 25-27, 2019. Please
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 TherapyA developing treatment that aims to fix, replace, or add genetic instructions. 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