Levodopa is not a useful treatment for Lesch-Nyhan disease

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

doi:10.1002/mds.23478

Levodopa is not a useful treatment for Lesch-Nyhan disease

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

School of Medicine

Research output: Contribution to journal › Article › peer-review

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.

Original language	English (US)
Pages (from-to)	746-749
Number of pages	4
Journal	Movement Disorders
Volume	26
Issue number	4
DOIs	https://doi.org/10.1002/mds.23478
State	Published - Mar 2011

Keywords

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

ASJC Scopus subject areas

Neurology
Clinical Neurology

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title = "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.",

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

Abstract

Keywords

ASJC Scopus subject areas

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