Trials for Cerebellar Ataxias : From Cellular Models to Human Therapies / edited by Bing-wen Soong, Mario Manto, Alexis Brice, Stefan M. Pulst.

Format:

Book

Author/Creator:

Soong, Bing-wen.

Contributor:

Manto, Mario.

Brice, Alexis.

Pulst, Stefan-M.

Series:

Contemporary Clinical Neuroscience, 2627-5341

Language:

English

Subjects (All):

Neurosciences.

Neurology.

Pharmaceutical chemistry.

Cognitive neuroscience.

Neuroscience.

Pharmaceutics.

Cognitive Neuroscience.

Local Subjects:

Neuroscience.

Neurology.

Pharmaceutics.

Cognitive Neuroscience.

Physical Description:

1 online resource (716 pages)

Edition:

1st ed. 2023.

Place of Publication:

Cham : Springer International Publishing : Imprint: Springer, 2023.

Summary:

Despite the critical importance of the cerebellum in brain functions, 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.

Contents:

Part I: Basic Science of Cerebellum and Ataxias

Functional anatomy of the cerebellum

Cerebellar physiology

Cerebellar biochemistry/pharmacology

Genetics of dominant ataxias

Autosomal and X-linked degenerative ataxias: from genetics to promising therapeutics

Seeking therapies for Spinocerebellar Ataxia: from gene silencing to systems-based approaches

Ion Channel Genes and Ataxia

Part II: Biomarkers and tools of trials

How to Design a Therapeutic Trial in SCAs

Therapy development for spinocerebellar ataxia: Rating Scales and Biomarkers

Clinical rating scales for ataxia

Scale for Ocular motor Disorders in Ataxia (SODA): Procedures and Basic Understanding

Cerebellar learning in the prism adaptation task

Blood and CSF biomarkers in autosomal dominant cerebellar ataxias

Part III: Autosomal Dominant Cerebellar Ataxias

Riluzole in progressive cerebellar ataxias

ASOs against ATXN2 in preclinical and phase 1 trials

Antisense oligonucleotide therapy against SCA3

Spinocerebellar ataxia type 7: From mechanistic pathways to therapeutic opportunities

Experimental neurotransplantation for cerebellar ataxias

Development of mesenchymal stem cells therapy for the treatment of polyglutamine SCA: from bench to bedside

Cerebello-Spinal tDCS as Rehabilitative Intervention in Neurodegenerative Ataxia

Cerebellar Transcranial Magnetic Stimulation in Cerebellar Ataxias

Physical therapy in Cerebellar Ataxia

Part IV: Autosomal Recessive Cerebellar Ataxias

Recent advances on therapeutic approaches for Friedreich’s Ataxia

Therapeutic use of interferon gamma in Friedreich ataxia

Metabolic treatments of cerebellar ataxia

Clinical Trials in Fragile X-Associated Tremor/Ataxia Syndrome

Part V: Sporadic Ataxias

Therapeutic strategies inimmune-mediated cerebellar ataxias

Coenzyme Q10 in MSA

State of the Art and History of Therapeutics in Ataxias.

Other Format:

Print version: Soong, Bing-wen Trials for Cerebellar Ataxias

ISBN:

3-031-24345-5