February 2, 2021
Spark Therapeutics Announces First Participant Dosed in Phase 1/2 Study of Investigational Gene Therapy for Late-Onset Pompe Disease
First participant dosed in the RESOLUTESM trial, a Phase 1/2 dose-escalation study of SPK-3006 Enrollment of approximately 20 total study
January 22, 2021
10th Annual PCMA Pull for Pompe!
The AMDA is excited to announce that the 10th Annual PCMA’s Pull for Pompe will take place on Saturday, April
January 14, 2021
Dr. Priya Kishnani, an Informative Session on the Covid 19 Vaccine with Questions and Answers
Webinar Details Title: Informative Session on the Covid 19 Vaccine with Questions and Answers Date: Thursday, January 14, 2021 Speaker:Dr.
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. More for gene therapyA developing treatment that aims to fix, replace, or add genetic instructions. More for Pompe.
July 26, 2020
Spark Therapeutics Clinical Trial FAQ
Spark Therapeutics has developed a “Frequently Asked Questions” document regarding their RESOLUTE clinical trialA research study that tests new treatments or approaches in people. More. 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