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Updated: Jun 2, 2026

Induction and Assessment of Levodopa-induced Dyskinesias in a Rat Model of Parkinson's Disease
Published on: October 14, 2021
Levodopa is not a useful treatment for Lesch-Nyhan disease
Jasper E Visser1, David J Schretlen, Bastiaan R Bloem
1Department of Neurology, Donders Institute for Brain, Cognition and Behavior, Radboud University Nijmegen Medical Centre, Nijmegen, The Netherlands. j.visser@neuro.umcn.nl
Abstract:
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.
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