Skip to main content
SpringerLink
Log in

Trials for Cerebellar Ataxias

From Cellular Models to Human Therapies

  • Book
  • © 2023

Overview

Editors:
  1. Bing-wen Soong

    1. Professor of Neurology, Shuang Ho Hospital, Taipei Medical University, Taipei, (Republic of China), Taiwan

    View editor publications

    You can also search for this editor in PubMed   Google Scholar

  2. Mario Manto

    1. Professor of Neuroanatomy, University of Mons and CHU-Charleroi, Charleroi, Belgium

    View editor publications

    You can also search for this editor in PubMed   Google Scholar

  3. Alexis Brice

    1. Paris Brain Institute, (Institut du Cerveau – ICM), CNRS INSERM, Sorbonne Université, APHP, Paris, France

    View editor publications

    You can also search for this editor in PubMed   Google Scholar

  4. Stefan M. Pulst

    1. Department of Neurology, Spencer Fox Eccles School of Medicine University of Utah, Salt Lake City, USA

    View editor publications

    You can also search for this editor in PubMed   Google Scholar

  • Provides the first book dedicated to ataxia trials
  • Offers a major step toward advancing the field by presenting effective therapeutics for these disorders
  • Presents comprehensive guidelines from the top experts in the field of cerebellar ataxias

Part of the book series: Contemporary Clinical Neuroscience (CCNE)

This is a preview of subscription content, log in via an institution to check access.

Access this book

Log in via an institution
eBook USD 189.00
Price excludes VAT (USA)
  • Available as EPUB and PDF
  • Read on any device
  • Instant download
  • Own it forever
Hardcover Book USD 249.99
Price excludes VAT (USA)
  • Durable hardcover edition
  • Dispatched in 3 to 5 business days
  • Free shipping worldwide - see info

Tax calculation will be finalised at checkout

Other ways to access

Licence this eBook for your library

Institutional subscriptions

Table of contents (29 chapters)

  1. Front Matter

    Pages i-xii
    Download chapter PDF

  2. Basic Science of Cerebellum and Ataxias

    1. Front Matter

      Pages 1-1
      Download chapter PDF

    2. Functional Anatomy of the Cerebellum

      • Izumi Sugihara, Yuanjun Luo, Richard Nana Abankwah Owusu-Mensah
      Pages 3-41

    3. Cerebellar Physiology

      • Jasmine Pickford, Richard Apps
      Pages 43-81

    4. Cerebellar Biochemistry/Pharmacology

      • Takahiro Seki
      Pages 83-114

    5. Genetics of Dominant Ataxias

      • Ashraf Yahia, Giovanni Stevanin
      Pages 115-139

    6. Autosomal and X-Linked Degenerative Ataxias: From Genetics to Promising Therapeutics

      • Anya Hadji, Aurélie Louit, Vincent Roy, Mathieu Blais, François Berthod, François Gros-Louis et al.
      Pages 141-181

    7. Seeking Therapies for Spinocerebellar Ataxia: From Gene Silencing to Systems-Based Approaches

      • Rachael Powers, Henry Paulson, Sharan Srinivasan
      Pages 183-207

    8. Ion Channel Genes and Ataxia

      • Mahesh Padmanaban, Christopher M. Gomez
      Pages 209-261

  3. Biomarkers and Tools of Trials

    1. Front Matter

      Pages 263-263
      Download chapter PDF

    2. How to Design a Therapeutic Trial in SCAs

      • Caterina Mariotti, Mario Fichera, Lorenzo Nanetti
      Pages 265-289

    3. Therapy Development for Spinocerebellar Ataxia: Rating Scales and Biomarkers

      • Chih-Chun Lin, Sheng-Han Kuo
      Pages 291-315

    4. Clinical Rating Scales for Ataxia

      • Tanja Schmitz-Hübsch
      Pages 317-345

    5. Scale for Ocular Motor Disorders in Ataxia (SODA): Procedures and Basic Understanding

      • Aasef G. Shaikh, Ji-Soo Kim, Caroline Froment, Yu Jin Koo, Nicolas Dupre, Marios Hadjivassiliou et al.
      Pages 347-361

    6. Cerebellar Learning in the Prism Adaptation Task

      • Takeru Honda, Hidehiro Mizusawa
      Pages 363-377

    7. Blood and CSF Biomarkers in Autosomal Dominant Cerebellar Ataxias

      • Giulia Coarelli, Alexandra Durr
      Pages 379-392

  4. Autosomal Dominant Cerebellar Ataxias

    1. Front Matter

      Pages 393-393
      Download chapter PDF

    2. Riluzole in Progressive Cerebellar Ataxias

      • Silvia Romano, Carmela Romano, Emanuele Morena, Fernanda Troili, Agnese Suppiej, Marco Salvetti et al.
      Pages 395-401

    3. ASOs Against ATXN2 in Preclinical and Phase 1 Trials

      • Stefan M. Pulst
      Pages 403-416

    4. Antisense Oligonucleotide Therapy Against SCA3

      • Hayley S. McLoughlin
      Pages 417-431
Back to top

Keywords

About this book

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.

Editors and Affiliations

  • Professor of Neurology, Shuang Ho Hospital, Taipei Medical University, Taipei, (Republic of China), Taiwan

    Bing-wen Soong

  • Professor of Neuroanatomy, University of Mons and CHU-Charleroi, Charleroi, Belgium

    Mario Manto

  • Paris Brain Institute, (Institut du Cerveau – ICM), CNRS INSERM, Sorbonne Université, APHP, Paris, France

    Alexis Brice

  • Department of Neurology, Spencer Fox Eccles School of Medicine University of Utah, Salt Lake City, USA

    Stefan M. Pulst

About the editors

Bing-wen Soong, MD, Ph.D.  is Professor in Neurology and Genetics (board-certified neurologist and geneticist, certified clinical trial investigator), Taipei Medical University and National Yang Ming Chiao Tung University Faculty of Medicine,  Taipei, Taiwan (Republic of China)



Mario Manto, MD, PhD, is the Head of the Department of Neurology at CHU-Charleroi in Belgium. He is also a Professeur de Neuroanatomie et Sémiologie Neurologique, Service des Neurosciences, at UMons, Mons, Belgium.



Alexis Brice, MD, Ph.D., is the Executive Director at the Paris Brain Institute in the ICM, Hôpital de la Pitié-Salpêtrière, Sorbonne Université  in France. 


Stefan-M. Pulst, M.D., Dr. med, is a Professor and  Chair in the Department of Neurology, at the University of Utah. 

Bibliographic Information

  • Book Title: Trials for Cerebellar Ataxias

  • Book Subtitle: From Cellular Models to Human Therapies

  • Editors: Bing-wen Soong, Mario Manto, Alexis Brice, Stefan M. Pulst

  • Series Title: Contemporary Clinical Neuroscience

  • DOI: https://doi.org/10.1007/978-3-031-24345-5

  • Publisher: Springer Cham

  • eBook Packages: Biomedical and Life Sciences, Biomedical and Life Sciences (R0)

  • Copyright Information: The Editor(s) (if applicable) and The Author(s), under exclusive license to Springer Nature Switzerland AG 2023

  • Hardcover ISBN: 978-3-031-24344-8Published: 30 May 2023

  • Softcover ISBN: 978-3-031-24347-9Due: 30 June 2023

  • eBook ISBN: 978-3-031-24345-5Published: 29 May 2023

  • Series ISSN: 2627-535X

  • Series E-ISSN: 2627-5341

  • Edition Number: 1

  • Number of Pages: XI, 727

  • Number of Illustrations: 1 b/w illustrations

  • Topics: Neurosciences, Neurology, Pharmaceutical Sciences/Technology, Neurosciences

Publish with us

Policies and ethics

Back to top

Search

Navigation