Neurodegenerative disorders: A clinical guide

Orla Hardiman, Julie A. Kelly, Thomas Bak, Marwa Elamin, Dragos L. Mihaila, Pamela J. Shaw, Hiroshi Mitsumoto, Jeremy M. Shefner

Research output: Chapter in Book/Report/Conference proceedingChapter


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.
Original languageEnglish
Title of host publicationNeurodegenerative Disorders
EditorsOrla Hardiman, P. Colin Doherty, Marwa Elamin, Peter Bede
Place of PublicationCham
PublisherSpringer International Publishing
Number of pages15
ISBN (Print)978-3-319-23309-3
Publication statusPublished - 27 Feb 2016


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