Levodopa is not a useful treatment for Lesch-Nyhan disease

Levodopa is not a useful treatment for Lesch-Nyhan disease

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

Movement Disorders

2011

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

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.

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