Charlotte Lawrenson, Martin Bares, Anita Kamondi, Andrea Kovács, Bridget Lumb, Richard Apps, Pavel Filip and Mario Manto; Cerebellum & Ataxias 20185:8 doi:10.1186/s40673-018-0087-9
Its dense connectivity with cerebral cortex, thalamic nuclei, brainstem nuclei and spinal cord, as well as its critically high number of neurons put the cerebellum in a unique position for a participation in cognitive, affective and sensorimotor operations. This special session has highlighted this aspect by taking fear behaviour, motor control, timing contributions and tremor as 4 examples of productive fields of research.
Friday, May 4, 2018
Current and Promising Therapies in Autosomal Recessive Ataxias
Vincent Picher-Martel, Nicolas Dupre. CNS Neurol Disord Drug Targets. 2018 Apr 18. doi: 10.2174/1871527317666180419115029. [Epub ahead of print]
The aim of this review is to provide a comprehensive clinical profile and to review the currently available therapies. We overview the physiopathology, neurological features and diagnostic approach of the common recessive ataxias. The emphasis is also made on potential drugs currently or soon-to-be in clinical trials. For instance, promising gene therapies raise the possibility of treating differently Friedreich’s ataxia, Ataxia-telangiectasia, Wilson’s disease and Niemann-Pick disease in the next few years.
The aim of this review is to provide a comprehensive clinical profile and to review the currently available therapies. We overview the physiopathology, neurological features and diagnostic approach of the common recessive ataxias. The emphasis is also made on potential drugs currently or soon-to-be in clinical trials. For instance, promising gene therapies raise the possibility of treating differently Friedreich’s ataxia, Ataxia-telangiectasia, Wilson’s disease and Niemann-Pick disease in the next few years.
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