Levodopa is not a useful treatment for Lesch-Nyhan disease

Jasper E. Visser, David J. Schretlen, Bastiaan R. Bloem, Hyder A. Jinnah

Research output: Contribution to journalArticlepeer-review


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.

Original languageEnglish (US)
Pages (from-to)746-749
Number of pages4
JournalMovement Disorders
Issue number4
StatePublished - Mar 2011


  • Dyskinesias
  • Dystonia
  • Lesch-Nyhan disease
  • Levodopa
  • Self-injurious behavior
  • Treatment

ASJC Scopus subject areas

  • Neurology
  • Clinical Neurology


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