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Wednesday, June 26 • 2:00pm - 3:15pm
#363: Neurodegenerative Diseases: Early-Stage Challenges and Optimal Models in Drug Development

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Component Type: Session
Level: Intermediate
CE: ACPE 1.25 Knowledge UAN: 0286-0000-19-687-L04-P; CME 1.25; IACET 1.25; RN 1.25

Intense research on the multiple fronts advanced our understanding of neurodegenerative diseases. Many disease-specific animal models have been used to test emergent medicines in neurology. Genetics and mechanism of neuronal pathogenesis contributed greatly and has created a wealth of knowledge and became the bases for novel technologies and multiple therapeutic targets for these neurodegenerative diseases. There is however substantial difficulty in choosing/accessing an optimal model or choosing measurements which would be truly informative of the product’s efficacy in neurodegenerative rare diseases or more prevalent diseases like pediatric neurodegenerative diseases and Alzheimer’s. This session will discuss nonclinical models supporting efficacy in rare neurodegenerative conditions, looking also at the challenges in early development in pediatric neurodegenerative diseases and highlight successes and failures in the development of treatments for dyslipidemia that may be applicable to the treatment of neurodegenerative diseases like Alzheimer’s disease.

Learning Objectives

Describe the importance and value of nonclinical in vivo models in rare neurodegenerative conditions and share highlights on the challenges in early development in pediatric neurodegenerative diseases; Identify optimal strategies for testing putative Alzheimer therapies; Recognize how to select and qualify biomarkers to identify patient and measure relevant drug activity; Define how to apply lessons from development of drugs to prevent Alzheimer Dementia.


Dinah Duarte, MSc


Rare Neurodegenerative Diseases: Early-Stage Challenges and Optimal Animal Models in Orphan Drug Development
Dinah Duarte, MSc

Alzheimers Disease Prophylaxis: Lessons from Lipid Therapeutics
Richard Scheyer, MD

The Challenges in Early Development in pediatric Neurodegenerative Diseases
Scott Demarest, MD


Scott Demarest

Assistant Professor, Departments of Pediatrics and Neurology, University of Colorado School of Medicine; Children's Hospital Colorado
Scott Demarest received a Bachelor of Science in biology from the University of Texas at Austin before going on to medical school at the University of Texas Health Science Center in San Antonio. He completed his residency in Pediatrics and child neurology at Children's National Health... Read More →
avatar for Dinah Duarte

Dinah Duarte

Scientific Evaluation Unit, Directorate of Medicinal Products, INFARMED
Dr. Dinah Duarte is a senior assessor at the Scientific Evaluation Unit at the Directorate of Medicinal Products, in the Portuguese regulatory authority for medicines and health products (INFARMED). She is an expert member at the European Medicines Agency (EMA); the current Committee... Read More →
avatar for Richard Scheyer

Richard Scheyer

Vice President, Medical, Medpace
Dr. Richard Scheyer is VP Medical at Medpace. Prior to Medpace, he led Experimental Medicine, BM, and PGx functions at Daiichi Sankyo. He served in leadership roles at Sanofi-Aventis and CMO at Neurotrope Bioscience. Dr. Scheyer received his BS Physics from Stanford, MD from SUNY... Read More →

Wednesday June 26, 2019 2:00pm - 3:15pm PDT
Room 2 San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA