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Past AMDA Webinars
The Common C.-32-13T G Splicing Variant of GAA Gene
Title: The common c.-32-13T>G splicingA normal step in how the body reads gene instructions. If splicing goes wrong, the wrong protein may be made. variant of GAA geneThe gene responsible for making the GAA enzyme.: from functional characterization to the identification of new therapeutic targets for Pompe diseaseA rare genetic disease in which the body cannot properly break down glycogen, leading to buildup that damages muscles and can affect breathing and, in some cases, the heart..
Speaker:
Andrea Dardis, PhD
Spark Therapeutics Gene Therapy Research Webinar
Title: Spark Therapeutics Gene TherapyA developing treatment that aims to fix, replace, or add genetic instructions. Research Webinar
Date: Tuesday, September 25, 2025
Speakers:
Federico Mingozzi, PhD – Chief Scientific Officer
Paul Monahan, MD – Translation Medicine Leader, Metabolic Disorders
Amy Fisher, MS, CGC – Patient Advocacy Lead
Webinar Overview:
Take this opportunity to learn more about Spark as a company and the field of gene therapy research.
Design And Performance Of Clinical Trials: An Overview
Title: Design and Performance of Clinical TrialsA research study that tests new treatments or approaches in people.: An Overview
Date: Monday, October 20, 2014
Speaker:
Anne Zajicek, MD, PharmD, FAAP, Chief, Obstetric and Pediatric, Pharmacology and Therapeutics Branch, National Institute of Child Health and Human Development (NICHD)