The Huntington's disease drug pipeline: a review of small molecules and their therapeutic targets.

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Tác giả: Shakir Ahamad, Nargis Bano, Shahnawaz Ali Bhat, Sameera Khan, Vladimir N Uversky

Ngôn ngữ: eng

Ký hiệu phân loại: 617.752 Eyeglasses, contact lenses, intraocular lenses

Thông tin xuất bản: Netherlands : Progress in molecular biology and translational science , 2025

Mô tả vật lý:

Bộ sưu tập: NCBI

ID: 55526

Huntington's disease (HD) is a progressive neurodegenerative condition resulting from a CAG repeat expansion in the huntingtin gene (HTT). Recent advancements in understanding HD's cellular and molecular pathways have paved the way for identifying various effective small-molecule candidates to treat the disorder. Two small molecules, Tetrabenazine and Deutetrabenazine, are approved for managing chorea associated with HD, and several others are under clinical trials. Notably, the field of small-molecule therapeutics targeting HD is rapidly progressing, and there is anticipation of their approval in the foreseeable future. This chapter provides a comprehensive overview of the emergence of small-molecule therapeutics in various stages of clinical development for HD therapy. The emphasis is placed on detailing their structural design, therapeutic effects, and specific mechanisms of action. Additionally, exploring key drivers implicated in HD pathogenesis offers valuable insights, as a foundational principle for designing prospective anti-HD therapeutic leads.
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