Oral pharmacological treatment of X-linked dystonia parkinsonism: successes and failures.

There is a paucity of published literature on the different oral medications tried for X-linked dystonia parkinsonism (XDP). In practice, most XDP patients are tried or have been tried on medications typically used for patients with generalized dystonia. These drugs include anticholinergic agents, baclofen, clonazepam and other benzodiazepines, tetrabenazine, and clozapine. Although several articles have shown that these classes of drugs are beneficial for patients with generalized dystonia, none have been systematically studied specifically for XDP patients. We are currently conducting the first randomized, placebo-controlled trial on the use of levodopa for the symptomatic treatment of XDP. This article reviews the data on the various dystonia medications that have been used in XDP.