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This workshop brings together leading healthcare professionals, clinician-scientists, translational researchers, and drug/therapy development experts with the primary aims of educating participants, building a cross-disciplinary understanding of therapeutic disease modification in DEEs and RGNDD, and empowering the next generation of young healthcare/scientific professionals entering the field.
 

Through interactive sessions led by internationally recognized experts, the program provides in-depth education on the scientific, clinical, ethical, and regulatory dimensions of drug development—from mechanism-driven discovery to clinical trial design, and long-term outcome assessment.

 

STEERING COMMITTEE

Stéphane Auvin, MD, PhD

Paris-Cité University & Robert-Debré University Hospital, Paris, France

 

Jacqueline French, MD, FAAN

Langone Health's Comprehensive Epilepsy Center

 

Emma James, DPhil, MFPM (Hon)

1. Encoded Therapeutics, 2. Pediatric Gene Therapy and Medical Ethics (PGTME) Working Group

 

Amy McTague, MBChB PhD

UCL Great Ormond Street Institute of Child Health, London, UK

 

Matthew Walker, PhD FRCP

National Hospital for Neurology and Neurosurgery, and UCL Queen Square Institute of Neurology, London, UK