Experimental Therapeutics for Dystonia
Identifieur interne : 000178 ( Ncbi/Checkpoint ); précédent : 000177; suivant : 000179Experimental Therapeutics for Dystonia
Auteurs : H Jinnah [États-Unis] ; Ellen Hess [États-Unis]Source :
- Neurotherapeutics : the journal of the American Society for Experimental NeuroTherapeutics [ 1933-7213 ] ; 2008.
Abstract
Dystonia is a neurological syndrome characterized by excessive involuntary muscle contractions leading to twisting movements and unnatural postures. It has many different clinical manifestations, and many different causes. More than 3 million people worldwide suffer from dystonia, yet there are few broadly effective treatments. In the past decade, progress in research has advanced our understanding of the pathogenesis of dystonia to a point where drug discovery efforts are now feasible. There are several strategies that can be used to develop novel therapeutics for dystonia. Existing therapies have only modest efficacy, but may be refined and improved to increase benefits while reducing side effects. Identifying rational targets for drug intervention based on the pathogenesis of dystonia is another strategy. The surge in both basic and clinical research discoveries has provided insights at all levels including etiological, physiological and nosological, to enable such a targeted approach. The empirical approach to drug discovery is complementary to the rational approach whereby compounds are identified using a non-mechanistic strategy. [MD1] With the recent development of multiple animal models of dystonia, it is now possible to develop assays and perform drug screens on vast number of compounds. This multifaceted approach to drug discovery in dystonia will likely provide lead compounds that can then be translated for clinical use.
Url:
DOI: 10.1016/j.nurt.2008.01.001
PubMed: 18394563
PubMed Central: 2322876
Affiliations:
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<front><div type="abstract" xml:lang="en"><p id="P2">Dystonia is a neurological syndrome characterized by excessive involuntary muscle contractions leading to twisting movements and unnatural postures. It has many different clinical manifestations, and many different causes. More than 3 million people worldwide suffer from dystonia, yet there are few broadly effective treatments. In the past decade, progress in research has advanced our understanding of the pathogenesis of dystonia to a point where drug discovery efforts are now feasible. There are several strategies that can be used to develop novel therapeutics for dystonia. Existing therapies have only modest efficacy, but may be refined and improved to increase benefits while reducing side effects. Identifying rational targets for drug intervention based on the pathogenesis of dystonia is another strategy. The surge in both basic and clinical research discoveries has provided insights at all levels including etiological, physiological and nosological, to enable such a targeted approach. The empirical approach to drug discovery is complementary to the rational approach whereby compounds are identified using a non-mechanistic strategy. [MD1] With the recent development of multiple animal models of dystonia, it is now possible to develop assays and perform drug screens on vast number of compounds. This multifaceted approach to drug discovery in dystonia will likely provide lead compounds that can then be translated for clinical use.</p>
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