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Levodopa is not a useful treatment for Lesch‐Nyhan disease
Author(s) -
Visser Jasper E.,
Schretlen David J.,
Bloem Bastiaan R.,
Jinnah Hyder A.
Publication year - 2011
Publication title -
movement disorders
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 3.352
H-Index - 198
eISSN - 1531-8257
pISSN - 0885-3185
DOI - 10.1002/mds.23478
Subject(s) - levodopa , dystonia , dopaminergic , disease , neuroscience , medicine , psychology , dopamine , cognition , movement disorders , physical medicine and rehabilitation , parkinson's disease
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. © 2011 Movement Disorder Society