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Pediatric Drug Development Workshop

Session 1: Developing Innovative Technologies for Children: Pediatric-Directed Drug Development

Session Chair(s)

Andrew  Mulberg, MD

Andrew Mulberg, MD

  • Senior Vice President
  • Neurogene, United States

This session will explore the innovative use of technologies in pediatric drug development. A presentation of scientific underpinnings of novel technology for the treatment of inborn errors of metabolism and CNS neurodegenerative diseases will be presented. Overcoming the technical challenges of gene therapy to the CNS, approaches for improved protein expression targeting, and stability for novel gene therapy. The presentations will be followed by a panel discussion.

Learning Objective :

At the conclusion of this session, participants should be able to:

  • understand the science and technology of novel gene transfer and therapies on evolution of potential cures for treatment of neurodegenerative diseases affecting children
  • develop an appreciation and understanding of the science underlying novel approaches to gene transfer in inborn errors of metabolism


James M Wilson, MD, PhD


James M Wilson, MD, PhD

  • University of Pennsylvania, United States
Kathrin  Meyer, DrSc, PhD


Kathrin Meyer, DrSc, PhD

  • Principal Investigator, Assistant Professor, The Ohio State University
  • Nationwide Children’s Hospital, United States
Cheryl  Yoder, RN


Cheryl Yoder, RN

  • Patient Advocate
  • Cure SMA, United States