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

Room 15B
Sponsored by the Division for Translational and Clinical Pharmacology (TCP)
Co-sponsored by the Division for Behavioral Pharmacology (BEH)
Co-sponsored by the Division for Drug Discovery and Development (DDD)
Co-sponsored by the Division for Drug Metabolism and Disposition (DMDD)
Co-sponsored by the Division for Molecular Pharmacology (MP)
Co-sponsored by the Division for Pharmacology Education (DPE)
Co-sponsored by Division for Toxicology (TOX)

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.

Regulatory Considerations in Orphan Drug Development
Eunjoo Pacifici - University of Southern California School of Pharmacy

Spinal Muscular Atrophy: An Overview from Genetics to Therapeutics
Karen Chen - Spinal Muscular Atrophy Foundation

Getting Rid of the Bad: HTT Lowering Therapeutics Development
Douglas Macdonald - CHDI Management / CHDI Foundation

Translating Breakthrough Science Discoveries into Novel Therapeutics for Rett Syndrome (RTT)
Randal Carpenter - RETT Syndrome Research Trust

Opportunities for Drug Development in Amyotrophic Laterals Sclerosis (ALS)
Lucie Bruijn - The ALS Association

AAV9 Based Gene Therapy for Neurological Disorders
Kathrin Meyer - Nationwide Children’s Hospital

Thank you to our Annual Meeting partners:

University of Florida
Wake Forest University
Washington State University College of Pharmacy and Pharmaceutical Sciences
University of Wisconsin Molecular and Cellular Pharmacology Training Program
UT Health San Antonio
University of Minnesota

Emory University Pharmacology

PR & P
University of Michigan Medical School Pharmacology