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Past AMDA Webinars
Lentiviral Gene Therapy for Pompe Disease
Title: Lentiviral 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
Date: Tuesday, October 10, 2023
Time: 10 AM CT, 11 AM ET
Speaker:
Dr. Pim Pijnappel, Associate Professor in Cell and Gene TherapyA developing treatment that aims to fix, replace, or add genetic instructions. More at the Erasmus MC University Medical Center, Rotterdam, the Netherlands
Webinar Overview:
The webinar will start with an introduction on Pompe diseaseA rare genetic disease in which the body cannot properly break down glycogen, leading to buildup tha... More with the latest information on what we know of the disease and what the current challenges are for treatments. Then, the concept of gene therapyA developing treatment that aims to fix, replace, or add genetic instructions. More will be explained as a possible new treatment option. The focus will be on lentiviral gene therapyA developing treatment that aims to fix, replace, or add genetic instructions. More, which is 1 of many possible forms of gene therapyA developing treatment that aims to fix, replace, or add genetic instructions. More. 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.
Spark Therapeutics Webinar: Trial Expectations
Title: Spark Therapeutics Webinar: Trail Expectations – What to Expect
Date: Wednesday, September 27, 2023
Time: 1 PM CT / 2 PM ET
Speaker:
Jacose Bell
Webinar Overview:
This presentation, developed by Spark Therapeutics, takes audience members through the introductory science of AAVA harmless virus commonly used as a delivery system in gene therapy to carry healthy genes into cell... More gene therapyA developing treatment that aims to fix, replace, or add genetic instructions. More. We will examine how AAVA harmless virus commonly used as a delivery system in gene therapy to carry healthy genes into cell... More theoretically works in a liver-directed approach, how that is different from other investigational forms of cell and gene therapyA developing treatment that aims to fix, replace, or add genetic instructions. More, and what the challenges and potential risks are with AAVA harmless virus commonly used as a delivery system in gene therapy to carry healthy genes into cell... More gene therapyA developing treatment that aims to fix, replace, or add genetic instructions. More. The presentation will then focus the remainder of its time on the role of clinical research and things people should consider if they are interested in participating in clinical research for investigational gene and cell therapies.
How to Manage LOPD Patients Diagnosed Through NBS
Title: New insights into Pompe diseaseA rare genetic disease in which the body cannot properly break down glycogen, leading to buildup tha... More since the advent of NBS
Date: Thursday, August 10, 2023
Time: 1 PM CT / 2 PM ET
Speakers:
Dr. Priya Kishnani, MD
Erin Huggins, MS, CGC
Webinar Overview:
An overview of the lessons learned from newborn screening for Pompe diseaseA rare genetic disease in which the body cannot properly break down glycogen, leading to buildup tha... More and its impact on disease management.