Abstract
Significant progress made over the last few decades in the management and treatment of many neurodegenerative diseases. However, successful translation of effective therapeutic compounds from animal models to humans has been disappointing. This chapter provides an overview of the current models of drug development and clinical trial design along with a summary of the advantages and limitations of each model. The chapter also discusses the challenges of performing clinical trials in neurodegenerative disorders, including the selection of appropriate biomarkers of disease activity and the appropriate definition of meaningful clinical outcomes, and identifies promising new approaches towards the development of efficacious disease modifying therapeutics.
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Hardiman, O. et al. (2016). Clinical Trials in Neurodegeneration. In: Hardiman, O., Doherty, C., Elamin, M., Bede, P. (eds) Neurodegenerative Disorders. Springer, Cham. https://doi.org/10.1007/978-3-319-23309-3_16
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DOI: https://doi.org/10.1007/978-3-319-23309-3_16
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