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Session 1: Developing Innovative Technologies for Children: Pediatric-Directed Drug Development
Session Chair(s)
Andrew Mulberg, MD
Senior Vice President, Neurogene, United States
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
Speaker(s)
Speaker
James M Wilson, MD, PhD
President and CEO, Founder, GEMMABio (Gemma Biotherapeutics), United States
Speaker
Kathrin Meyer, DRSc, PhD
Principal Investigator, Assistant Professor, The Ohio State University, Nationwide Children’s Hospital, United States
Panelist
Cheryl Yoder, RN
Patient Advocate, Cure SMA, United States
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