The neurobiological basis for novel experimental therapeutics in dystonia

Anthony M. Downs, Kaitlyn M. Roman, Simone A. Campbell, Antonio Pisani, Ellen J. Hess, P. Bonsi

Research output: Contribution to journalReview articlepeer-review


Dystonia is a movement disorder characterized by involuntary muscle contractions, twisting movements, and abnormal postures that may affect one or multiple body regions. Dystonia is the third most common movement disorder after Parkinson's disease and essential tremor. Despite its relative frequency, small molecule therapeutics for dystonia are limited. Development of new therapeutics is further hampered by the heterogeneity of both clinical symptoms and etiologies in dystonia. Recent advances in both animal and cell-based models have helped clarify divergent etiologies in dystonia and have facilitated the identification of new therapeutic targets. Advances in medicinal chemistry have also made available novel compounds for testing in biochemical, physiological, and behavioral models of dystonia. Here, we briefly review motor circuit anatomy and the anatomical and functional abnormalities in dystonia. We then discuss recently identified therapeutic targets in dystonia based on recent preclinical animal studies and clinical trials investigating novel therapeutics.

Original languageEnglish
Article number104526
JournalNeurobiology of Disease
Publication statusPublished - Oct 1 2019


  • Anatomy
  • Animal models
  • Basal ganglia
  • Cerebellum
  • Drug discovery
  • Therapy

ASJC Scopus subject areas

  • Neurology


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