Title: The Pompe Survey: Past Insights and Future Goals
Date: Tuesday, March 25, 2025
Time: 11 AM CT / 12 PM ET
Speakers:
Lauren Dobischok, MSc
Michelle Kruijshaar, PhD
Webinar Overview:
Launched in 2002, the IPA/Erasmus MC Pompe Survey collects information on the impacts of Pompe disease on patient’s lives, and how these impacts may change with treatment. Pompe patients from around the world provide this information through an annual questionnaire. The information collected in the Pompe Survey provides insights on the effect of different treatments and particular issues that Pompe patients may face in daily life. With second-generation ERT entering the market and other novel therapies on the horizon, insights from patients’ perspectives are more relevant than ever. Learn about the history of the Pompe Survey, key findings from the survey, and the continued importance of patient participation in achieving future goals in this webinar.

Title: Evolving our understanding of Pompe Disease – 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.

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!

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 therapy approaches for late-onset Pompe disease (LOPD) currently under evaluation in clinical trials, 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.