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Novel Approaches for Accessing the CNS: Nonclinical and Clinical Challenges
Session Chair(s)
Richard Scheyer, MD
Vice President, Medical
Medpace, United States
This session will highlight new approaches in CNS-targeted therapeutics. These approaches to identify and develop therapies able to reach the CNS and address specific genetic perturbations are being pioneered in rare disease, and require disciplined approaches to study designs and execution.
Learning Objective : Design studies for advanced treatment modalities, with focus on CNS therapeutics. Identify and address hurdles in pre-clinical evaluation, identification of appropriate patients, and administration of treatments crossing or bypassing the blood brain barrier: Describe target engagement and biologic impact.
Speaker(s)
Nonclinical Models Supporting Orphan Drug Designations in Rare Neurodegenerative Conditions
Dinah Duarte, MSC
European Medicines Agency, Netherlands
Signal Management Lead
Novel Central Nervous System Delivery Methods in the Era of Targeted Therapeutics
William Elmquist, PHARMD, PHD
Univerity of Minnesota, United States
Director, Brain Barriers Research; Distinguished Prof, Dept. of Pharmaceutics
Novel Approaches to Confirming CNS Penetration and Target Engagement
Richard Scheyer, MD
Medpace, United States
Vice President, Medical