Dystonia is a recognised neurological feature of Wilson's disease that requires systematic pharmacological management. When the initial treatment step fails to achieve adequate reduction and control of dystonic symptoms, an escalation to a structured next-line regimen is indicated.
The established first-line approach uses botulinum toxin A (BTX) injections for focal dystonia, targeting the most affected muscles, or oral anticholinergics — trihexyphenidyl or biperiden — as initial oral therapy for broader or generalised involvement. The treatment goal at this line is reduction and control of dystonic symptoms. When that goal is not met, the next step is required.
After failure of first-line agents, oral medications drawn from several distinct pharmacological classes are available as the next step. The full protocol — including agent selection, sequencing, and monitoring — is detailed in the structured regimen.
DOI: 10.1016/j.jhep.2024.11.007
Focal dystonias are usually effectively treated with BTX injections and segmental or generalized dystonias are treated with oral drugs alone or in combination with BTX injected into the most affected muscles.
Other pharmacological agents include (a) baclofen (ranging between 60 and 120 mg/day), (b) benzodiazepines (c) levodopa or dopamine agonists, (d) tetrabenazine (starting dose 12.5 mg, increased up to 50–75 mg/day), and (e) carbamazepine (up to 900 mg/day) or oxcarbazepine (up to 300 mg/day).
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