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 March 09, 2020, was postponed. It has been moved to a Virtual Meeting that will take place Monday, July 13, 2020, from...
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 Pull For Pompe Shoot due to the on-going COVID-19 pandemic. When we initially postponed the Shoot to August 15, we had high hopes that the virus would...
AMDA Q and A with Dr. Priya Kishnani on COVID-19 and Pompe
With all of the questions and uncertainty surrounding the ongoing COVID-19 pandemic, the AMDA worked with Dr. Priya Kishnani and her group at Duke University on the following Question and Answer Fact Sheet. We hope that the Pompe Community will find the information...
Statement from Spark Therapeutics on COVID-19 and their Pompe Program
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 COVID-19 (SARS-CoV-2) outbreak. Given this serious health situation, the US Centers for Disease Control and Prevention (CDC)...
Statement from Amicus regarding COVID-19
Please click here to read a statement from Amicus Therapeutics regarding COVID-19.
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 PCMA Pull for Pompe has been postponed until Saturday, August 15, 2020. The Team of volunteers for The Pull For Pompe...
Upcoming Webinar: January 23, 2020
AMDA WEBINAR Title: The common c.-32-13T>G splicing variant of GAA gene: from functional characterization to the identification of new therapeutic targets for Pompe disease. Date: January 23, 2020 Time: 9:30am PT, 11:30am CT, 12:30pm ET Register Now: ...
2019 AMDA/IPA Conference Report Available
On October 25-27, 2019, almost 200 Pompe patients and their families, scientists, doctors, and industry representatives from around the world gathered in San Antonio, Texas for the 2019 AMDA/IPA Pompe Patient and Scientific Conference. Over the course of this...
Patient Focused Drug Development Meeting to be held March 09, 2020
The AMDA is excited to announce that it is partnering with the MDA to host a Patient Focused Drug Development Meeting (PFDD) on March 09, 2020. The PFDD meeting is an opportunity for patients and families living with Pompe disease to share their perspective and inform...
2019 Helen Walker Research Grant For Pompe Disease Now Accepting Applications
The AMDA is excited to announce a research grant opportunity for applicants who are involved in Pompe Disease research. This grant opportunity was made possible by the 2019 Pull for Pompe, a private fundraiser held by the Precast Manufacturer’s Association of Texas...
2019 AMDA/IPA International Conference
The AMDA/IPA International Pompe Patient and Scientific Conference will take place in San Antonio, Texas from October 25-27, 2019. Please click here for the 2019 Conference Registration Form. Each family, speaker, patient organization representative, industry...
2019 PCMA’s Pull For Pompe!
The AMDA is excited to announce that the 2019 PCMA’s Pull for Pompe will take place on Saturday, April 13th at the National Shooting Complex in San Antonio, Texas! This is the 9th Annual Pull for Pompe hosted by the PCMA. The Pull for Pompe clay shooting event is...
Press Release: Amicus Therapeutics Announces First Patient Dosed in Phase 3 PROPEL Study
Amicus Therapeutics Announces First Patient Dosed in Phase 3 PROPEL Pivotal Study of AT-GAA in Patients with Pompe Disease Global Study to Assess AT-GAA Compared to Standard-of-Care Enzyme Replacement Therapy (ERT) over 52 Weeks CRANBURY, N.J., Dec. 20, 2018— Amicus...
Spark Therapeutics Gene Therapy Research Webinar
Spark Therapeutics Gene Therapy Research Webinar Download an audio only version
Press Release: Spark Therapeutics Announces New Preclinical Data for Pompe Disease Gene Therapy
Spark Therapeutics Announces New Preclinical Data for Pompe Disease Gene Therapy Candidate Data from IND-enabling studies in three species support moving SPK-3006 into the clinic in 2019 PHILADELPHIA, Oct. 08, 2018—Spark Therapeutics, a fully integrated, commercial...
AMDA Webinar: Spark Therapeutics Gene Therapy Research
SPARK THERAPEUTICS GENE THERAPY RESEARCH WEBINAR Please join the AMDA for a webinar focused on Spark Therapeutics and gene therapy research Take this opportunity to learn more about Spark as a company and the field of gene therapy research. We will be joined by...
2018 Helen Walker Research Grant For Pompe Disease Now Accepting Applications
The AMDA is excited to announce a research grant opportunity for applicants who are involved in Pompe Disease research. This grant opportunity was made possible by the 2018 Pull for Pompe, a private fundraiser held by the Precast Manufacturer’s Association of Texas...
Press Release: Amicus Announces Regulatory and Clinical Updates for AT-GAA in Pompe Disease
CRANBURY, N.J., Sept. 10, 2018—Amicus Therapeutics, a global biotechnology company focused on discovering, developing and delivering novel medicines for rare metabolic diseases, announced today regulatory and clinical advancements in its development program AT-GAA for...
Press Release from Erasmus Medical Center: Making Muscle from Skin
Researchers from the Erasmus MC in Rotterdam, Netherlands, have succeeded in generating muscle cells from skin cells. This is significant as skin cells can be relatively easily obtained from a skin biopsy. The team developed a protocol to obtain pure muscle cells (ie...
2017 Helen Walker Research Grant For Pompe Disease Now Accepting Applications
The AMDA is excited to announce a research grant opportunity for applicants who are involved in Pompe Disease research. This grant opportunity was made possible by the 2017 Pull for Pompe, a private fundraiser held by the Precast Manufacturer’s Association of Texas...