First participant dosed in the RESOLUTESM trial, a Phase 1/2 dose-escalation study of SPK-3006 Enrollment of approximately 20 total study
The AMDA is excited to announce that the 10th Annual PCMA’s Pull for Pompe will take place on Saturday, April
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 read.
As everyone knows, due to the ongoing COVID-19 pandemic, the Patient-Focused Drug Development Meeting (PFDD) that was originally scheduled for
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
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 organizers; the 2020
AMDA WEBINAR Title: The common c.-32-13T>G splicing variant of GAA gene: from functional characterization to the identification of new therapeutic
The AMDA is excited to announce that it is partnering with the MDA to host a Patient Focused Drug Development
The AMDA is excited to announce a research grant opportunity for applicants who are involved in Pompe Disease research. This
The AMDA is excited to announce that the 2019 PCMA’s Pull for Pompe will take place on Saturday, April 13th
Amicus Therapeutics Announces First Patient Dosed in Phase 3 PROPEL Pivotal Study of AT-GAA in Patients with Pompe Disease Global
Spark Therapeutics Announces New Preclinical Data for Pompe Disease Gene Therapy Candidate Data from IND-enabling studies in three species support
SPARK THERAPEUTICS GENE THERAPY RESEARCH WEBINAR Please join the AMDA for a webinar focused on Spark Therapeutics and gene therapy
The AMDA is excited to announce a research grant opportunity for applicants who are involved in Pompe Disease research. This
CRANBURY, N.J., Sept. 10, 2018—Amicus Therapeutics, a global biotechnology company focused on discovering, developing and delivering novel medicines for rare
Researchers from the Erasmus MC in Rotterdam, Netherlands, have succeeded in generating muscle cells from skin cells. This is significant