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The Emerging Landscape of Small-Molecule Therapeutics for the Treatment of Huntington’s Disease

9 Dec 2022

Abstract excerpt

Huntington's disease (HD) is a progressive neurodegenerative disorder caused by a CAG repeat expansion in the huntingtin gene (HTT). The new insights into HD's cellular and molecular pathways have led to the identification of numerous potent small-molecule therapeutics for HD therapy. The field of HD-targeting small-molecule therapeutics is accelerating, and the approval of these therapeutics to combat HD may be...

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PMID: 36490325
DOI: 10.1021/acs.jmedchem.2c00799

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