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020 _a9783031243455
024 7 _a10.1007/978-3-031-24345-5
_2doi
040 _aES-MaUEC
_bspa
_cES-MaUEC
_dES-MaUEC
050 4 _aRC376.5
_b2023 EB
245 0 0 _aTrials for Cerebellar Ataxias :
_bFrom Cellular Models to Human Therapies
_cedited by Bing-wen Soong, Mario Manto, Alexis Brice, Stefan M Pulst
250 _a1st ed. 2023
264 1 _aCham
_bSpringer International Publishing
_c2023
300 _a1 recurso en línea
336 _atexto
_btxt
_2rdacontent
337 _aelectrónico
_bc
_2rdamedia
338 _arecurso electrónico
_bcr
_2rdacarrier
490 0 _aContemporary Clinical Neuroscience
_x2627-5341
505 0 _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.
520 _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.
988 _aSpringer_BiomedLife_2023
650 7 _2embne
_9148577
_aTrastornos motores
650 7 _2embne
_9188115
_aSistema nervioso central
_xEnfermedades
856 4 0 _uhttps://go.openathens.net/redirector/universidadeuropea.es?url=https://doi.org/10.1007/978-3-031-24345-5
_zAcceso a este recurso digital (usuarios Universidad Europea de Madrid)
942 _2lcc
_cLE
998 _b06/2024
_dz
_eIG
_zSI