TOP NEWS
Closing the Gaps: Pompe Disease Newborn Screening and the Importance of RUSP Alignment
A Milestone for Pompe Families This SeptemberSeptember is Newborn Screening Awareness Month, a time to celebrate the programs that ensure every baby has a healthy start. This year, there's especially good news for families affected by Pompe disease: on August 18,...
OTHER NEWS
Aro Biotherapeutics to Present New Preclinical Data
Aro Biotherapeutics to Present New Preclinical Data Highlighting the Potential of Centyrin-siRNA Conjugates for the Treatment of Pompe Disease. To read the article, please click here.
2022 PCMA Pull for Pompe Fundraiser
The AMDA is excited to announce that the 11th Annual PCMA’s Pull for Pompe will take place on Saturday, April 30, 2022 at the National Shooting Complex in San Antonio, Texas! The Pull for Pompe clay shooting event is designed for all ages and ability levels. Please...
2021 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 2021 Pull for Pompe, a private fundraiser held by the Precast Manufacturer’s Association of Texas...
Re-Scheduled–Friday, Oct 8, 2021: AMDA and Spark Webinar: Understanding Gene Therapy Research and the Potential Applications for Late Onset Pompe Disease
Hi All, We are excited to announce that the webinar is on for Friday, October, 8, 2021 at 1 PM CST. If you have already registered, you don't need to do anything. If you have NOT registered yet, it's not too late! Please click here to register. And, don't forget, we...
FDA approves Sanofi’s Nexviazyme® (avalglucosidase alfa-ngpt) for Late-Onset Pompe
PARIS – August 6, 2021 - The U.S. Food and Drug Administration (FDA) has approved Nexviazyme® (avalglucosidase alfa-ngpt) for the treatment of patients one year of age and older with late-onset Pompe disease, a progressive and debilitating muscle disorder that impairs...
February 2021 Amicus’ AT-GAA Shows Clinically Meaningful & Significant Improvements in Both Musculoskeletal and Respiratory Measures
Amicus’ AT-GAA Shows Clinically Meaningful & Significant Improvements in Both Musculoskeletal and Respiratory Measures in Late-Onset Pompe Disease Compared to Standard of Care in Pivotal Phase 3 PROPEL Study. Amicus Therapeutics, Inc. Download file Download...
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 participants is ongoing PHILADELPHIA, February 1, 2021 (GLOBE NEWSWIRE)— Spark Therapeutics, a member of the Roche Group (SIX: RO,...
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 4, 2020 at the National Shooting Complex in San Antonio, Texas! The Pull for Pompe clay shooting event is designed for all ages and ability levels. Please...
Audentes Pompe Program Update
Audentes has released a Pompe Program Update. It includes information regarding FORTIS, their clinical trial for gene therapy for Pompe. To read the Pompe Program Update, please click here.
Spark Therapeutics Clinical Trial FAQ
Spark Therapeutics has developed a “Frequently Asked Questions” document regarding their RESOLUTE clinical trial. Please click here to read.
Acid Maltase Deficiency Association
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