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.

 Current and Promising Therapies in Autosomal Recessive Ataxias