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 disease to share their perspective and inform...
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...