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Neurodegenerative Diseases: Early-Stage Challenges and Optimal Models in Drug Development
Session Chair(s)
Dinah Duarte, MSc
Signal Management Lead
European Medicines Agency, Netherlands
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 Objective : 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.
Speaker(s)
Rare Neurodegenerative Diseases: Early-Stage Challenges and Optimal Animal Models in Orphan Drug Development
Dinah Duarte, MSc
European Medicines Agency, Netherlands
Signal Management Lead
Alzheimers Disease Prophylaxis: Lessons from Lipid Therapeutics
Richard Scheyer, MD
Medpace, United States
Vice President, Medical
The Challenges in Early Development in pediatric Neurodegenerative Diseases
Scott Demarest, MD
University of Colorado School of Medicine; Children's Hospital Colorado, United States
Assistant Professor, Departments of Pediatrics and Neurology
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