Aquatic Physical Therapy Webinar
Title: Aquatic Exercise: Applications for Pompe Disease
Date: August 26, 2022
Time: 1:30 PM CT / 2:30 PM ET
Speaker:
Dr. Kendra Lucas, PT, DPT, Aquatic Physical Therapist at Kettering Health, Ohio
Webinar Overview:
The unique properties of water provide surprising benefits for individuals struggling with gait difficulty, muscle weakness, cardiorespiratory impairments, balance problems and other symptoms associated with Pompe disease. Aquatic physical therapy offers a safe alternative to land-based programs. The water’s buoyancy off-loads joint pressure, allowing you more freedom to move with less energy exertion. Join Dr. Lucas to learn much more about how aquatic exercise can help you stay active and healthy so you can live your best life.
Astellas Gene Therapies Press Release – Clinical Trial Update
"On June 26, 2022, Astellas Pharma, Inc. ("Astellas") issued a press release announcing that the U.S. Food and Drug Administration (FDA) has placed a clinical hold on the FORTIS Phase 1/2 clinical trial evaluating AT845 ("the investigational gene therapy candidate")...
Voice of the Patient Report for the Pompe PFDD is Released!
The AMDA is excited to report that the final Voice of the Patient Report for the Pompe Patient-Focused Drug Development meeting has been released. To view the report, please CLICK HERE!
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.
AMDA Zoom Webinar With Sanofi
Title: POMPE REGISTRY: 18 YEARS OF LEADERSHIP AND CONTRIBUTIONS
Date: Monday, April 11, 2022
Speaker:
Joseph Bender, MD, MBA, Global Head of Rare Disease Registries
Danielle Dong, ScM, CGC, Global Operations and Advocacy Lead Rare Disease Registries
Webinar Overview:
The Sanofi Genzyme Pompe Registry is a global, observational, and voluntary program designed to track natural history and outcomes of patients diagnosed with Pompe disease.
*After multiple conversations with Sanofi, the AMDA regrets to inform the Community that the recording of the recent AMDA Webinar on the Sanofi Pompe Registry will be removed from the AMDA website on May 31, 2022.
However, as a result of these conversations, the AMDA is pleased to say that Sanofi has committed to developing educational material regarding the Registry. We hope that this will be available soon, and will keep the Community posted on developments.
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...
AMDA and Spark Webinar: Understanding Gene Therapy Research and the Potential Applications for Late Onset Pompe Disease
To watch the Webinar, please click here!
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...
September 2020 Pregnancy Outcomes in Late-Onset Pompe Disease
Pregnancy Outcomes in Late-Onset Pompe Disease Life Download file
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...
Phenotypic implications of pathogenic variant types in Pompe disease
Manuel A. Viamonte1,2 ● Stephanie L. Filipp3 ● Zara Zaidi4,8 ● Matthew J. Gurka3 ● Barry J. Byrne4 ● Peter B. Kang 1,5,6,7 Received: 25 January 2021 / Revised: 1 April 2021 / Accepted: 20 April 2021 © The Author(s), under exclusive licence to The Japan Society of...
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...
Dr. Priya Kishnani, an Informative Session on the Covid 19 Vaccine with Questions and Answers
Title: Informative Session on the Covid 19 Vaccine with Questions and Answers
Date: Thursday, January 14, 2021
Speaker:
Dr. Priya Kishnani
National Patient-Teacher Registry: A New Educational Concept
Title: National Patient-Teacher Registry: A New Educational Concept
Date: Wednesday, December 9, 2020
Speaker:
Mark Korson, PhD, VMP Genetics, LLC, Director of Physician Support, Director of Education
Webinar Overview:
In general, medical students and post-graduate trainees receive little teaching about metabolic disease. At VMP Genetics, we believe in the power of “patient-teaching” and are bringing patients and families into lectures and presentations – at conferences and in the classroom. While doctors talk facts, patients tell stories. Story-telling is a more compelling teaching method with better recall over time than didactic lecturing. We also believe that doctors are more likely to make a diagnosis if they have already seen a patient and hear her/his story. We are looking to collaborate in this effort with patient foundations and similar communitites.
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.
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...