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Updated: Jul 6, 2025

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Levodopa for Dystonia in Children: A Case Series and Review of the Literature
Alexandra Lesenskyj DeArias1, Nigel S Bamford2
1Department of Pediatrics, Yale University, New Haven, Connecticut; Department of Neurology, Yale University, New Haven, Connecticut.
Background:
Levodopa is used to treat hyperkinetic movements in children with dopa-responsive dystonia. However, levodopa may also be helpful in treating other forms of dystonia when used beyond a brief trial period.
Methods:
We performed a retrospective review of all children referred to our institution for evaluation of generalized dystonia and subsequently treated with carbidopa-levodopa. Motor function was assessed using video recordings and examination notes, quantified with the Burke-Fahn-Marsden Dystonia Rating Scale.
Results:
Long-term treatment with carbidopa-levodopa moderately improved motor function, whereas short-term use did not. Carbidopa-levodopa was well tolerated without untoward effects.
Conclusions:
Dystonia is a significant cause of disability with limited effective treatment options. Published work is restricted but generally supports the findings of this review. A well-controlled study to examine the utility of carbidopa-levodopa treatment for dystonia is needed.
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