Levodopa is not a useful treatment for Lesch-Nyhan disease
behavior problems; tone and motor problems; Lesch-Nyhan syndrome; pharmacologic intervention; levodopa; levodopa-dopa; carbidopa
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.Copyright © 2011 Movement Disorder Society.
Bloem B R; Schretlen D J; Jinnah H A; Visser J E
Movement Disorders
2011
Article information provided for research and reference use only. PedPalASCNET does not hold any rights over the resource listed here. All rights are retained by the journal listed under publisher and/or the creator(s).
<a href="http://doi.org/10.1002/mds.23478" target="_blank" rel="noreferrer noopener">10.1002/mds.23478</a>
A favorable response to antiparkinsonian treatment in juvenile neuronal ceroid lipofuscinosis
tone and motor problems; NCL3; NCL6; pharmacologic intervention; levodopa; selegiline
To study the effect of dopaminergic drugs on the parkinsonism in juvenile neuronal ceroid lipofuscinosis, the authors conducted an open study of 21 patients. According to the motor Unified PD Rating Scale (UPDRS) score, treatment was initiated with either levodopa (n = 10) or selegiline (n = 6). Five patients served as a control group. The UPDRS score after 1 year was compared with the score at onset. Both in the control group and in the selegiline group, the mean UPDRS score increased, whereas in the levodopa group, the mean UPDRS score decreased. The difference between the levodopa group and the control group was significant.
Aberg L E; Rinne J O; Rajantie I; Santavuori P
Neurology
2001
Article information provided for research and reference use only. PedPalASCNET does not hold any rights over the resource listed here. All rights are retained by the journal listed under publisher and/or the creator(s).
<a href="http://doi.org/10.1212/WNL.56.9.1236" target="_blank" rel="noreferrer noopener">10.1212/WNL.56.9.1236</a>