Levodopa is not a useful treatment for Lesch-Nyhan disease

Title

Levodopa is not a useful treatment for Lesch-Nyhan disease

Creator

Bloem B R; Schretlen D J; Jinnah H A; Visser J E

Identifier

Publisher

Movement Disorders

Date

2011

Subject

behavior problems; tone and motor problems; Lesch-Nyhan syndrome; pharmacologic intervention; levodopa; levodopa-dopa; carbidopa

Description

Lesch-Nyhan disease (LND) is characterized by dystonia, cognitive abnormalities, and self-injurious behavior. No effective therapies are available. LND is associated with a presynaptic dopaminergic deficit, but the reported effects of dopamine replacement therapy are conflicting. The current prospective open-label study assesses the effects of levodopa on both neurological and behavioral features of LND. All 6 study participants discontinued levodopa early, due to lack of effect and sometimes worsening of motor function. The results provide important clues for pathophysiological mechanisms and suggestions for future treatment options.Copyright © 2011 Movement Disorder Society.

Rights

Article information provided for research and reference use only. PedPalASCNET does not hold any rights over the resource listed here. All rights are retained by the journal listed under publisher and/or the creator(s).

Citation

Bloem B R; Schretlen D J; Jinnah H A; Visser J E, “Levodopa is not a useful treatment for Lesch-Nyhan disease,” Pediatric Palliative Care Library, accessed August 3, 2021, https://pedpalascnetlibrary.omeka.net/items/show/16590.

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