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Past AMDA Webinars
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.
Lentiviral Gene Therapy for Pompe Disease
Title: Lentiviral gene therapy for Pompe disease
Date: Tuesday, October 10, 2023
Time: 10 AM CT, 11 AM ET
Speaker:
Dr. Pim Pijnappel, Associate Professor in Cell and Gene Therapy at the Erasmus MC University Medical Center, Rotterdam, the Netherlands
Webinar Overview:
The webinar will start with an introduction on Pompe disease with the latest information on what we know of the disease and what the current challenges are for treatments. Then, the concept of gene therapy will be explained as a possible new treatment option. The focus will be on lentiviral gene therapy, which is 1 of many possible forms of gene therapy. Recent results from the laboratory will be presented, followed by an update on next steps toward clinical development. The current landscape of new therapies for rare diseases will be discussed, including the need to develop novel therapies for reasonable and transparent prices.