Trials for Cerebellar Ataxias: From Cellular Models to Human Therapies

Soong, Bing-wen; Manto, Mario; Brice, Alexis; Pulst, Stefan M.

Despite the critical importance of the cerebellum in brain function, the scientific community still lacks effective treatments for most cerebellar ataxias.  This book provides  a link between the pathogenesis and therapies of cerebellar ataxias while also providing a comprehensive assessment of the preclinical and clinical trials dedicated to cerebellar ataxias over the past 20 years of progress.  This is the first book fully dedicated to the trials and therapies of these disorders. It is a truly authoritative and comprehensive reference, and comes at a time of major advances in genetic tools and neuroimaging assessments.  The coverage begins by laying a foundation of the basic science of the cerebellum and ataxias, proceeds to discuss biomarkers and the tools of trials, offers guidelines on conducting trials,  and then explores the full range of therapeutics and their trials, including gene therapy and cell transplantation. The authors are top experts on cerebellar research and the contributing authors have all made seminal contributions in the field.

Details

Published by: Springer

Publication Date: 2024-05-30

Format: Paperback

ISBN-13: 9783031243479

DOI: 10.1007/978-3-031-24345-5

Dimensions: 235.0cm x155.0cm

Pages: 727.0