Main description:

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.

Contents:

Part I: Basic Science of Cerebellum and Ataxias 1 Functional anatomy of the cerebellum 2 Cerebellar physiology 3 Cerebellar biochemistry/pharmacology 4 Genetics of dominant ataxias 5 Autosomal and X-linked degenerative ataxias: from genetics to promising therapeutics 6 Seeking therapies for Spinocerebellar Ataxia: from gene silencing to systems-based approaches 7 Ion Channel Genes and Ataxia Part II: Biomarkers and tools of trials 8 How to Design a Therapeutic Trial in SCAs 9 Therapy development for spinocerebellar ataxia: Rating Scales and Biomarkers 10 Clinical rating scales for ataxia 11 Scale for Ocular motor Disorders in Ataxia (SODA): Procedures and Basic Understanding 12 Cerebellar learning in the prism adaptation task 13 Blood and CSF biomarkers in autosomal dominant cerebellar ataxias Part III: Autosomal Dominant Cerebellar Ataxias 14 Riluzole in progressive cerebellar ataxias 15 ASOs against ATXN2 in preclinical and phase 1 trials 16 Antisense oligonucleotide therapy against SCA3 17 Spinocerebellar ataxia type 7: From mechanistic pathways to therapeutic opportunities 18 Experimental neurotransplantation for cerebellar ataxias 19 Development of mesenchymal stem cells therapy for the treatment of polyglutamine SCA: from bench to bedside 20 Cerebello-Spinal tDCS as Rehabilitative Intervention in Neurodegenerative Ataxia 21 Cerebellar Transcranial Magnetic Stimulation in Cerebellar Ataxias 22 Physical therapy in Cerebellar Ataxia Part IV: Autosomal Recessive Cerebellar Ataxias 23 Recent advances on therapeutic approaches for Friedreich's Ataxia 24 Therapeutic use of interferon gamma in Friedreich ataxia 25 Metabolic treatments of cerebellar ataxia 26 Clinical Trials in Fragile X-Associated Tremor/Ataxia Syndrome Part V: Sporadic Ataxias 27 Therapeutic strategies in immune-mediated cerebellar ataxias 28 Coenzyme Q10 in MSA 29 State of the Art and History of Therapeutics in Ataxias