Dopa-responsive Dystonia in Children.

Treatment of dopa-responsive dystonia is one of the more satisfying experiences in clinical neurology. The response to treatment with levodopa is usually dramatic and complete with no long-term complications. Carbidopa/levodopa is the mainstay in treating dopa-responsive dystonia. There is some experience using anticholinergic agents, but they are more likely to cause side effects and do not treat the underlying biochemical abnormality. Dopa-responsive dystonia caused by guanosine triphosphate cyclohydrolase I deficiency typically presents with dystonia in the lower extremities in the first decade of life. However, the presenting symptoms can vary. Thus, it is this author's recommendation that any child with dystonia receive a trial of carbidopa/levodopa.