The AMDA is excited to announce that the 10th Annual PCMA’s Pull for Pompe will take place on Saturday, April 4, 2020 at the National...

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

Spark Therapeutics has developed a “Frequently Asked Questions” document regarding their RESOLUTE clinical trial. Please click here to...

As everyone knows, due to the ongoing COVID-19 pandemic, the Patient-Focused Drug Development Meeting (PFDD) that was originally scheduled...

Update: July 8, 2020 It is with great sadness and disappointment that we hereby announce the cancellation of the 2020 Pull For Pompe Shoot...

With all of the questions and uncertainty surrounding the ongoing COVID-19 pandemic, the AMDA worked with Dr. Priya Kishnani and her group...

Dear IPA Board of Directors, As I’m sure you are aware, the World Health Organization (WHO) has declared a pandemic in response to the...

Please click here to read a statement from Amicus Therapeutics regarding COVID-19.

Announcement Regarding COVID-19 and the Pull for Pompe (03/18/2020): After careful consideration, and long discussions among the...

AMDA WEBINAR Title:  The common c.-32-13T>G splicing variant of GAA gene: from functional characterization to the identification of new...