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      Therapeutic approaches to Huntington disease: from the bench to the clinic

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      Nature Reviews Drug Discovery
      Springer Nature America, Inc

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          Abstract

          <p class="first" id="d271936e97">The 25 years since the identification of the gene responsible for Huntington disease (HD) have stood witness to profound discoveries about the nature of the disease and its pathogenesis. Despite this progress, however, the development of disease-modifying therapies has thus far been slow. Preclinical validation of the therapeutic potential of disrupted pathways in HD has led to the advancement of pharmacological agents, both novel and repurposed, for clinical evaluation. The most promising therapeutic approaches include huntingtin (HTT) lowering and modification as well as modulation of neuroinflammation and synaptic transmission. With clinical trials for many of these approaches imminent or currently ongoing, the coming years are promising not only for HD but also for more prevalent neurodegenerative disorders, such as Alzheimer and Parkinson disease, in which many of these pathways have been similarly implicated. </p>

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          Author and article information

          Journal
          Nature Reviews Drug Discovery
          Nat Rev Drug Discov
          Springer Nature America, Inc
          1474-1776
          1474-1784
          September 21 2018
          September 21 2018
          September 21 2018
          September 21 2018
          Article
          10.1038/nrd.2018.133
          30237454
          b6838d24-b967-4bed-9324-d63c522a6dfc
          © 2018
          History

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