Check Back Soon for Upcoming Webinars

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.

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)

Title: The contribution of the IPA survey to our understanding of the natural course of Pompe disease and effects of enzyme therapy: Ten years of the IPA survey
Date: Thursday, February 27, 2014
Speaker:
J.C. van der Meijden, Center for Lysosomal and Metabolic diseases, Erasmus MC University Medical Center, Rotterdam, the Netherlands
Webinar Overview:
For more than ten years Pompe patients, from all over the world, participate in the International Pompe Association / Erasmus MC Pompe survey on a yearly basis. During this period we have gathered a vast amount of information on the natural course of Pompe disease and the effects of enzyme replacement therapy. During this webinar we will discuss what we have learned about Pompe disease by working with the Survey. Furthermore, we will discuss the importance of the survey and goals for the future. At the last part of the webinar there is an opportunity for participants to ask questions to the researchers.