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  • Title: Drug discovery and development for Huntington's disease - an orphan indication with high medical need.
    Author: Heitz F, La Rosa S, Gonzalez-Couto E, Gaviraghi G, Terstappen GC.
    Journal: IDrugs; 2008 Sep; 11(9):653-60. PubMed ID: 18763216.
    Abstract:
    Huntington's disease (HD) is a rare neurodegenerative disorder that progressively destroys the mental capacity and motor control of patients. This loss of motor control results in abnormal body movements (chorea) - the hallmark of HD. Given that no disease-modifying therapy for HD exists and that available symptomatic treatments are not highly efficacious, the medical need for this 'orphan' disease remains high. The number of compounds that are undergoing discovery and development for the treatment of HD has increased significantly in recent years, spurred by legislative incentives for orphan drug development and by support from non-profit foundations. Thus, hope exists for patients with HD that efficacious medicines will become available.
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