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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 diseaseA rare genetic disease in which the body cannot properly break down glycogen, leading to buildup tha... More – clinical trialA research study that tests new treatments or approaches in people. More 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. More approaches for late-onset Pompe disease (LOPD)A form of Pompe disease that begins after infancy and usually progresses more slowly. More currently under evaluation in clinical trialsA research study that tests new treatments or approaches in people. More, including liver-directed, central nervous system (CNS)-directed, and muscle-directed. The speakers will explain how clinical trialsA research study that tests new treatments or approaches in people. More help determine whether a potential gene therapyA developing treatment that aims to fix, replace, or add genetic instructions. More is a viable treatment option for people living with LOPDA form of Pompe disease that begins after infancy and usually progresses more slowly. More. The will also help the audience understand the goals of clinical trialsA research study that tests new treatments or approaches in people. More and the assessments that measure those goals.
Ask Bio Webinar: Development of Gene Therapy for Pompe Disease
Title: Development of Gene TherapyA developing treatment that aims to fix, replace, or add genetic instructions. More for Pompe DiseaseA rare genetic disease in which the body cannot properly break down glycogen, leading to buildup tha... More: 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 therapyA developing treatment that aims to fix, replace, or add genetic instructions. More for Pompe diseaseA rare genetic disease in which the body cannot properly break down glycogen, leading to buildup tha... More. He will provide a summary of research focused on clinical development of gene therapyA developing treatment that aims to fix, replace, or add genetic instructions. More with a focus on potential risks versus benefits, as well as expectations regarding the clinical use of gene therapyA developing treatment that aims to fix, replace, or add genetic instructions. More.
Boston Childrens Hospital Webinar: Defining Central Nervous System Abnormalities
Title: Defining Central Nervous System Abnormalities in Infantile and Late-Onset Pompe DiseaseA form of Pompe disease that begins after infancy and usually progresses more slowly. More 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)A form of Pompe disease that begins after infancy and usually progresses more slowly. More. We will describe our comprehensive approach toward understanding the neurobiological abnormalities in IOPDA severe form of Pompe disease that begins in infancy and often affects the heart and muscles. More/LOPD. Finally, we will share preliminary findings from ongoing studies involving patients with IOPDA severe form of Pompe disease that begins in infancy and often affects the heart and muscles. More/LOPD and that are taking place at Boston Children’s Hospital.