10th Annual PCMA Pull for Pompe!

Announcement Regarding COVID-19 and the Pull for Pompe (03/18/2020): After careful consideration, and long discussions among the organizers; the 2020 PCMA Pull for Pompe has been postponed until Saturday, August 15, 2020. The Team of volunteers for The Pull For Pompe...

Upcoming Webinar: January 23, 2020

AMDA WEBINAR 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... More variant of GAA geneThe gene responsible for making the GAA enzyme. More: from functional...

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 gathered in San Antonio, Texas for the 2019 AMDA/IPA Pompe Patient and Scientific Conference.  Over the course of this...

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 Meeting (PFDD) on March 09, 2020. The PFDD meeting is an opportunity for patients and families living with Pompe diseaseA rare genetic disease in which the...
The Common C.-32-13T G Splicing Variant of GAA Gene

The Common C.-32-13T G Splicing Variant of GAA Gene

Webinar Details Title: The common c.-32-13T>G splicing variant of GAA gene: from functional characterization to the identification of new therapeutic targets for Pompe disease. Speaker:Andrea Dardis, PhD **Due to a family emergency, Dr. Dardis was unable to present...

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 click here for the 2019 Conference Registration Form.  Each family, speaker, patient organization representative, industry...

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 at the National Shooting Complex in San Antonio, Texas! This is the 9th Annual Pull for Pompe hosted by the PCMA. The Pull for Pompe clay shooting event is...