July 27, 2020
Audentes Pompe Program Update
Audentes has released a Pompe Program Update. It includes information regarding FORTIS, their clinical trial for gene therapy for Pompe.
July 26, 2020
Spark Therapeutics Clinical Trial FAQ
Spark Therapeutics has developed a “Frequently Asked Questions” document regarding their RESOLUTE clinical trial. Please click here to read.
July 10, 2020
Patient Focused Drug Development Meeting—UPDATE
As everyone knows, due to the ongoing COVID-19 pandemic, the Patient-Focused Drug Development Meeting (PFDD) that was originally scheduled for
July 8, 2020
2020 PCMA Pull For Pompe!—Cancelled
Update: July 8, 2020 It is with great sadness and disappointment that we hereby announce the cancellation of the 2020
With all of the questions and uncertainty surrounding the ongoing COVID-19 pandemic, the AMDA worked with Dr. Priya Kishnani and