Handbook of the Cerebellum and Cerebellar Disorders pp 2370-2394 | Cite as
Novel Therapeutic Challenges in Cerebellar Diseases
In the last decade, substantial scientific progress has enabled a better understanding of the pathogenesis of cerebellar diseases and the improvement of their diagnoses. Extensive preclinical work is expanding the possibilities of using experimental models to analyze disease-specific mechanisms and to approach candidate therapeutic strategies to create a rationale for clinical trials that might finally lead to successful treatment. At present, drug treatment of cerebellar disorders has shown limited effectiveness and current treatment is primarily supportive. Until effective and selective pharmacological treatment leading to better quality of life as well as increased survival of patients with cerebellar diseases is found, physical and sensory rehabilitation techniques are revealing effective approaches for improving the patient’s quality of life. The objective of this chapter is to provide an updated summary of the treatments currently available for cerebellar disorders, in particular for spinocerebellar ataxias, and to discuss the new emerging therapeutic strategies that are resulting from the intensive ongoing basic and translational research devoted to cerebellar diseases.
KeywordsPhytanic Acid Spinal Muscular Atrophy Spinocerebellar Ataxia International Cooperative Ataxia Rate Scale Episodic Ataxia
Dr. Ivelisse Sanchez’s helpful comments and suggestions are kindly acknowledged. Dr. Antoni Matilla’s scientific research on ataxias is funded by the Spanish Ministry of Science and Innovation (BFU2008-00527/BMC), the Carlos III Health Institute (CP08/00027), the Latin American Science and Technology Development Programme (CYTED) (210RT0390), the European Commission (EUROSCA project, LHSM-CT-2004-503304), and the Fundació de la Marató de TV3 (Televisió de Catalunya). We are indebted to the Spanish Ataxia Association (FEDAES), the Spanish Federation for Rare Diseases (FEDER), and the ataxia patients for their continuous support and motivation. Antoni Matilla is a Miguel Servet Investigator in Neurosciences of the Spanish National Health System.
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