Highlighting advances in the discovery and development of new drug therapies for neurodegenerative disorders, this title focuses on the many aspects of the research on drugs to treat Huntington's disease. It also examines the practical realities of preclinical testing, clinical testing strategies, and, ultimately, clinical usage.
Drawing on the expertise of key researchers in the field, the book discusses the basic neurobiology of Huntington's disease and how its monogenic nature confers enormous practical advantages for translational research.
The book is written lucidly by authorities in their respective fields, covering clinical features, pathogenic mechanisms, protein interactions, preclinical models, biomarkers, small molecules and other approaches (eg, recombinant antibodies), screening strategies, and drug development. Informative figures and tables are provided, and reproduction of key figures as a set of colour plates provides a useful centrepiece. One unifying message is that the polyglutamine xpansion leads to a complex cascade of diverse molecular and cellular events, the progress of which is difficult to slow or halt. Innovative approaches described, such as the target validation process of the Cure Huntington's Disease Initiative (CHDI) Foundation, will be of interest to those studying other neurological diseases.--Anthony J Hannan, writing in The Lancet Neurology, March 2011