Audentes Pompe Program Update

Posted on: July 27, 2020 – Filed under

Audentes has released a Pompe Program Update. It includes information regarding FORTIS, their clinical trial for gene therapy for Pompe.

To read the Pompe Program Update, please click here.


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 splicing variant of GAA gene: from functional characterization to the identification of new therapeutic targets for Pompe disease. Date:  January 23, 2020 Time:  9:30am PT, 11:30am CT, 12:30pm ET Register Now: ...

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...

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...

AMDA Webinar: Spark Therapeutics Gene Therapy Research

SPARK THERAPEUTICS GENE THERAPY RESEARCH WEBINAR Please join the AMDA for a webinar focused on Spark Therapeutics and gene therapy research Take this opportunity to learn more about Spark as a company and the field of gene therapy research. We will be joined by...

 Acid Maltase Deficiency Association


Your generous contribution will go a long way into improving the quality of life of Pompe Disease Patients worldwide. Donations go towards Pompe Disease Research.

If you are interested in learning more about Pompe Disease and would like to make a contribution in support of necessary research, please contact us at:

(210) 494-6144


PO Box 700248

San Antonio, Texas 78270 USA