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.

SUBMITTER: Visser JE 

PROVIDER: S-EPMC3523804 | biostudies-literature | 2011 Mar

REPOSITORIES: biostudies-literature