New approaches to discovering drugs that treat dystonia.

: Dystonia consists of involuntary movements, abnormal posturing, and pain. In adults, dystonia presents in a particular region of the body and causes significant disability due to pain as well as impairment in activities of daily living and employment. The current gold standard treatment, botulinum toxin (BoNT), has limitations - painful, frequent injections due to 'wearing off' of treatment effect; expense; and expected side effects like swallowing difficulty and weakness. There is a clear therapeutic gap in our current treatment options for dystonia and also a clear need for an effective novel treatment. Testing any novel treatment is complicated because most adults with focal dystonia are treated with BoNT. : This review focuses on establishing the need for novel therapeutics. It also suggests potential leads from preclinical studies; and, discusses the issue of clinical trial readiness in the dystonia field. : Identifying a novel therapeutic intervention for dystonia patients faces two major challenges. The first is acknowledging the therapeutic gap that currently exists. Second, shifting some of our research aims in dystonia to clinical trial readiness is imperative if we are to be ready to test novel therapeutic agents.