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Past AMDA Webinars
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 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.
Ask Bio Webinar: Development of Gene Therapy for Pompe Disease
Title: Development of Gene Therapy for Pompe Disease: Recent History
Date: Tuesday, November 7, 2023
Time: 1 PM CT / 2 PM ET
Speaker:
Dr. Dwight Koeberl
Webinar Overview:
Dr. Koeberl will discuss the rationale for gene therapy for Pompe disease. He will provide a summary of research focused on clinical development of gene therapy with a focus on potential risks versus benefits, as well as expectations regarding the clinical use of gene therapy.
Boston Childrens Hospital Webinar: Defining Central Nervous System Abnormalities
Title: Defining Central Nervous System Abnormalities in Infantile and Late-Onset Pompe Disease Patients
Date: Tuesday, October 17, 2023
Time: 1 PM CT / 2 PM ET
Speakers:
Raquel van Gool
Jaymin Upadhyay
Webinar Overview:
In this presentation, we will provide a short description of neurological signs and symptoms that have been reported in patients with Infantile- or Late-Onset Pompe Disease (LOPD). We will describe our comprehensive approach toward understanding the neurobiological abnormalities in IOPD/LOPD. Finally, we will share preliminary findings from ongoing studies involving patients with IOPD/LOPD and that are taking place at Boston Children’s Hospital.