Levodopa is not a useful treatment for Lesch-Nyhan disease.

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.

• Authors: Jasper E Visser, David J Schretlen, Bastiaan R Bloem, Hyder A Jinnah
• Journal: Movement disorders : official journal of the Movement Disorder Society (Mov Disord) Vol. 26 Issue 4 Pg. 746-9 (Mar 2011) ISSN: 1531-8257 [Electronic] United States
• PMID: 21506156 (Publication Type: Journal Article, Research Support, N.I.H., Extramural, Research Support, Non-U.S. Gov't)
• Copyright: Copyright © 2011 Movement Disorder Society.

Chemical References

• Antiparkinson Agents
• Levodopa

Topics

• Adult
• Antiparkinson Agents
• Child
• Child, Preschool
• Contraindications
• Female
• Humans
• Lesch-Nyhan Syndrome (drug therapy)
• Levodopa
• Male
• Treatment Outcome