Mexiletine is an antiarrhythmic drug that has been reported to exert antidy
stonic properties. We performed an open-label study to collect further evid
ence of the antidystonic effect of mexiletine in spasmodic torticollis (ST)
and to evaluate its possible use in generalized dystonia. We administered
mexiletine to six patients with dystonia (three with generalized dystonia a
nd three with ST) who had failed to respond to previous pharmacotherapy. Th
e drug was started at a dose of 200 mg/d by mouth and increased up to a max
imum dose of 800 mg/d. Patients were evaluated at regular intervals over a
6-week period with use of the Fahn & Marsden Dystonia Scale and the Toronto
Western Spasmodic Torticollis Rating Scale (TWSTRS) and videotaped. At the
end of the trial, the videotapes were reviewed and scored by a blind obser
ver. Patients were then followed for at least 1 year and evaluated every 3
months at the dose reached during the study period. No adverse effects were
reported in Five patients; in one patient, dizziness developed at the dosa
ge of 800 mg/d, requiring a reduction of the dose. At the end of a 6-week p
eriod, a significant improvement in the rating scale for dystonia and in vi
deotape ratings was observed after mexiletine treatment (p < 0.01). Our dat
a indicate that mexiletine is a useful drug in dystonia treatment.