In This Section

Challenges and Promises of CNS Orphan Drug Development: Stories from Bench to Clinic

Tuesday April 24, 2018

9:30 am - 12:00 pm Eastern Time (ET)

Room 15B


Chair :

Daryl Davies
University of Southern California School of Pharmacy

Jeffrey Paul
jpharm consulting

Approximately 40% of new drugs approved in the last three years were approved for rare or orphan diseases. This symposium will inform the audience of the challenges, promises, and lessons learned from orphan disease investigations. We will focus on how preclinical drug development findings translated into clinical opportunities. Topics to be covered include an overview of regulatory challenges and benefits associated with orphan drug development followed by presentations on Spinal Muscular Atrophy, Huntington Disease, RETT syndrome, Amyotrophic Laterals Sclerosis. Presentations will include examples of successful translation of animal models to recently approved therapies and lessons learned along the way.


Eunjoo Pacifici - University of Southern California School of Pharmacy

Regulatory Considerations in Orphan Drug Development

Karen Chen - Spinal Muscular Atrophy Foundation

Spinal Muscular Atrophy: An Overview from Genetics to Therapeutics

Douglas Macdonald - CHDI Management / CHDI Foundation

Getting Rid of the Bad: HTT Lowering Therapeutics Development

Randal Carpenter - RETT Syndrome Research Trust

Translating Breakthrough Science Discoveries into Novel Therapeutics for Rett Syndrome (RTT)

Lucie Bruijn - The ALS Association

Opportunities for Drug Development in Amyotrophic Laterals Sclerosis (ALS)

Kathrin Meyer - Nationwide Children’s Hospital

AAV9 Based Gene Therapy for Neurological Disorders