December 9, 2020
National Patient-Teacher Registry: A New Educational Concept
Webinar Details Title: National Patient-Teacher Registry: A New Educational Concept Date: Wednesday, December 9, 2020 Speaker:Mark Korson, PhD, VMP Genetics,
July 27, 2020
Audentes Pompe Program Update
Audentes has released a Pompe Program Update. It includes information regarding FORTIS, their clinical trialA research study that tests new treatments or approaches in people. for gene therapyA developing treatment that aims to fix, replace, or add genetic instructions. 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
Dear IPA Board of Directors, As I’m sure you are aware, the World Health Organization (WHO) has declared a pandemic
March 19, 2020
Statement from Amicus regarding COVID-19
Please click here to read a statement from Amicus Therapeutics regarding COVID-19.
March 18, 2020
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
January 10, 2020
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 be made. variant of GAA geneThe gene responsible for making the GAA enzyme.: from functional characterization to the identification of new therapeutic