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Past AMDA Webinars
Evolving Our Understanding of Pompe Disease – Data Driven Insights
Title: Evolving our understanding of Pompe DiseaseA rare genetic disease in which the body cannot properly break down glycogen, leading to buildup that damages muscles and can affect breathing and, in some cases, the heart. – data driven insights
Date: Wednesday, February 26, 2025
Time: 1 PM CT / 2 PM ET
Speaker:
Ryan Colburn
Webinar Overview:
An overview of how NBS enabled significant updates to Pompe epidemiology (Prevalence, etc), and how the strength of this data enables an improved foundation for evolving our understanding of Pompe around the world, and across the full spectrum of disease. We’ll also cover how this foundation relates to other important topics, including some previously covered in this webinar series such as: ABC’s of Pompe, Disease Management in the NBS era, Treatment development, Emerging research, etc.
The ABCs of Pompe Disease
Title: The ABCs of Pompe Disease
Date: Monday, January 13, 2025
Time: 1 PM CT / 2 PM ET
Speaker:
Erin Huggins, MS, CGC
Webinar Overview:
Are you looking for information on Pompe disease or trying to navigate a new diagnosis? Overwhelmed by the amount of information online and the sea of jargon and terminology? Whether you are dealing with a diagnosis, are a family member or a caregiver, or even a medical provider who just wants a refresher on the basics, this webinar is for you! We will cover the “ABCs” of Pompe disease including its underlying cause, genetics/inheritance, newborn screening, treatment/management, and more. Information will be presented in a thorough, easy-to-follow manner and there will be time for questions at the end. We hope you’ll join us!
Astellas Gene Therapies Webinar: Evaluating therapies for Pompe disease – clinical trial goals and assessments
Title: Evaluating gene therapies for Pompe disease – clinical trial goals and assessments
Date: Monday, February 26, 2024
Speaker:
Jordi Díaz-Manera, MD, PhD
Christine S. Brown
Mark Walzer, PhD
Webinar Overview:
This Astellas Gene Therapies presentation provides an overview of the different gene therapyA developing treatment that aims to fix, replace, or add genetic instructions. approaches for late-onset Pompe disease (LOPD)A form of Pompe disease that begins after infancy and usually progresses more slowly. currently under evaluation in clinical trialsA research study that tests new treatments or approaches in people., including liver-directed, central nervous system (CNS)-directed, and muscle-directed. The speakers will explain how clinical trials help determine whether a potential gene therapy is a viable treatment option for people living with LOPD. The will also help the audience understand the goals of clinical trials and the assessments that measure those goals.