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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 splicing variant of GAA gene: from functional characterization to the identification of new therapeutic targets for Pompe disease.
Speaker:
Andrea Dardis, PhD
Spark Therapeutics Gene Therapy Research Webinar
Title: Spark Therapeutics Gene Therapy 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 Trials: 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)
