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Session 3: Leveraging Innovative Trials Design I: Product Development-Centric Registries and Platforms
Session Chair(s)
Edward Connor, MD, MS
Professor Emeritus Pediatrics, Microbiology, Immunology, Tropical Medicine
George Washington University School of Medicine and Health Sciences, United States
Andrew Mulberg, MD
Senior Vice President
Neurogene, United States
This session will explore the innovative use of trial designs and registry development to address clinical issues including understanding natural history and outcome assessment for effective endpoint development in pediatric drug development. A presentation of scientific underpinnings of effective and existing registry development for the treatment of pediatric diseases will be presented.
Speaker(s)
Speaker
Laura E Schanberg, MD
Duke University Medical Center, United States
Associate Professor and Co-chief, Pediatric Rheumatology
Speaker
John-Michael Sauer, PhD
Critical Path Institute, United States
Biomarkers Program Officer and Executive Director
Speaker
Abby Bronson, MBA
Edgewise Therapeutics, United States
Vice President, Patient Advocacy and External Innovation