TOP NEWS
Texas Adds Pompe Disease to Newborn Screening Panel—A Historic Milestone Coinciding with the AMDA’s 30th Anniversary
Great news for the rare disease community—Texas has taken a big step by adding Pompe disease to its newborn screening panel! This major advancement in protecting the health of Texas infants also marks a meaningful moment for the Acid Maltase Deficiency Association...
OTHER NEWS
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: ...
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 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...
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...
Acid Maltase Deficiency Association
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