LEADER 05227nam 22007095 450 001 9910728385703321 005 20230529101424.0 010 $a3-031-24345-5 024 7 $a10.1007/978-3-031-24345-5 035 $a(MiAaPQ)EBC30554457 035 $a(Au-PeEL)EBL30554457 035 $a(DE-He213)978-3-031-24345-5 035 $a(BIP)086557983 035 $a(PPN)270613927 035 $a(CKB)26801506900041 035 $a(EXLCZ)9926801506900041 100 $a20230529d2023 u| 0 101 0 $aeng 135 $aurcnu|||||||| 181 $ctxt$2rdacontent 182 $cc$2rdamedia 183 $acr$2rdacarrier 200 10$aTrials for Cerebellar Ataxias $eFrom Cellular Models to Human Therapies /$fedited by Bing-wen Soong, Mario Manto, Alexis Brice, Stefan M. Pulst 205 $a1st ed. 2023. 210 1$aCham :$cSpringer International Publishing :$cImprint: Springer,$d2023. 215 $a1 online resource (716 pages) 225 1 $aContemporary Clinical Neuroscience,$x2627-5341 311 08$aPrint version: Soong, Bing-wen Trials for Cerebellar Ataxias Cham : Springer International Publishing AG,c2023 9783031243448 327 $aPart 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 in immune-mediated cerebellar ataxias -- Coenzyme Q10 in MSA -- State of the Art and History of Therapeutics in Ataxias. 330 $aDespite the critical importance of the cerebellum in brain functions, the scientific community still lacks effective treatments for most cerebellar ataxias. This book provides a link between the pathogenesis and therapies of cerebellar ataxias while also providing a comprehensive assessment of the preclinical and clinical trials dedicated to cerebellar ataxias over the past 20 years of progress. This is the first book fully dedicated to the trials and therapies of these disorders. It is a truly authoritative and comprehensive reference and comes at a time of major advances in genetic tools and neuroimaging assessments. The coverage begins by laying a foundation of the basic science of the cerebellum and ataxias, proceeds to discuss biomarkers and the tools of trials, offers guidelines on conducting trials, and then explores the full range of therapeutics and their trials, including gene therapy and cell transplantation. The authors are top experts on cerebellar research and the contributing authors have all made seminal contributions in the field. 410 0$aContemporary Clinical Neuroscience,$x2627-5341 606 $aNeurosciences 606 $aNeurology 606 $aPharmaceutical chemistry 606 $aCognitive neuroscience 606 $aNeuroscience 606 $aNeurology 606 $aPharmaceutics 606 $aCognitive Neuroscience 610 $aInternal Medicine 610 $aMedical 615 0$aNeurosciences. 615 0$aNeurology. 615 0$aPharmaceutical chemistry. 615 0$aCognitive neuroscience. 615 14$aNeuroscience. 615 24$aNeurology. 615 24$aPharmaceutics. 615 24$aCognitive Neuroscience. 676 $a616.842 676 $a616.842 700 $aSoong$b Bing-wen$01362814 701 $aManto$b Mario$01056538 701 $aBrice$b Alexis$01362815 701 $aPulst$b Stefan-M$01362816 801 0$bMiAaPQ 801 1$bMiAaPQ 801 2$bMiAaPQ 906 $aBOOK 912 $a9910728385703321 996 $aTrials for Cerebellar Ataxias$93382346 997 $aUNINA