An overview of the pharmacotherapeutics for dystonia: advances over the past decade

Dystonia is the third most common movement disorder, impacting quality of life and work productivity. Its pathogenesis is heterogeneous, and many pharmacotherapeutics have been suggested for its treatment. The mainstay of management for focal dystonia is botulinum toxin. Oral pharmacotherapeutics usually are nonspecific and associated with risk of unwanted side effects such as drowsiness and lethargy. There is tremendous need for robust clinical trials for new pharmacotherapeutics as we deepen our understanding of dystonia.

This review will focus on the advances and research in the pharmacologic treatment of dystonia from January 2012 to April 2022. We performed a systematic database search on PubMed for the period mentioned.

Botulinum toxins remain the mainstay of focal dystonia treatment but may be insufficient for treatment of patients with more widespread dystonia manifestations. The most novel emerging therapies include daxibotulinumtoxinA, dipraglurant, and sodium oxybate. There is a strong clinical need for more effective therapeutic options in dystonia, which may involve the development of individualized treatment options based on dystonia subtype, etiology, or novel mechanisms of action that target specific underlying contributing features.