Document Detail


Levodopa is not a useful treatment for Lesch-Nyhan disease.
MedLine Citation:
PMID:  21506156     Owner:  NLM     Status:  MEDLINE    
Abstract/OtherAbstract:
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
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Publication Detail:
Type:  Journal Article; Research Support, N.I.H., Extramural; Research Support, Non-U.S. Gov't     Date:  2011-01-31
Journal Detail:
Title:  Movement disorders : official journal of the Movement Disorder Society     Volume:  26     ISSN:  1531-8257     ISO Abbreviation:  Mov. Disord.     Publication Date:  2011 Mar 
Date Detail:
Created Date:  2011-04-20     Completed Date:  2011-08-15     Revised Date:  2013-05-24    
Medline Journal Info:
Nlm Unique ID:  8610688     Medline TA:  Mov Disord     Country:  United States    
Other Details:
Languages:  eng     Pagination:  746-9     Citation Subset:  IM    
Copyright Information:
Copyright © 2011 Movement Disorder Society.
Affiliation:
Department of Neurology, Donders Institute for Brain, Cognition and Behavior, Radboud University Nijmegen Medical Centre, Nijmegen, The Netherlands. j.visser@neuro.umcn.nl
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MeSH Terms
Descriptor/Qualifier:
Adult
Antiparkinson Agents / contraindications*
Child
Child, Preschool
Female
Humans
Lesch-Nyhan Syndrome / drug therapy*
Levodopa / contraindications*
Male
Treatment Outcome
Grant Support
ID/Acronym/Agency:
HD053312/HD/NICHD NIH HHS; R01 HD053312/HD/NICHD NIH HHS
Chemical
Reg. No./Substance:
0/Antiparkinson Agents; 0/Levodopa
Comments/Corrections

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