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Pediatric Drug Development Workshop

Session 3: Leveraging Innovative Trials Design I: Product Development-Centric Registries and Platforms

Session Chair(s)

Edward  Connor, MD, MS

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

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.


Laura E Schanberg, MD


Laura E Schanberg, MD

  • Associate Professor and Co-chief, Pediatric Rheumatology
  • Duke University Medical Center, United States
John-Michael  Sauer, PhD


John-Michael Sauer, PhD

  • Biomarkers Program Officer and Executive Director
  • Critical Path Institute, United States
Abby  Bronson, MBA


Abby Bronson, MBA

  • Vice President, Patient Advocacy and External Innovation
  • Edgewise Therapeutics, United States