Huntington's disease is a chronic progressive neurodegenerative condition for which there is no disease-modifying treatment. The known genetic cause of Huntington's disease makes it possible to identify individuals destined to develop the disease and instigate treatments before the onset of symptoms. Multiple trials are already underway that target the cause of HD, yet clinical measures are often insensitive to change over typical clinical trial duration. Robust biomarkers of drug target engagement, disease severity and progression are required to evaluate the efficacy of treatments and concerted efforts are underway to achieve this. Biofluid biomarkers have potential advantages of direct quantification of biological processes at the molecular level, whilst imaging biomarkers can quantify related changes at a structural level in the brain. The most robust biofluid and imaging biomarkers can offer complementary information, providing a more comprehensive evaluation of disease stage and progression to inform clinical trial design and endpoints.

中文翻译：

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亨廷顿病的液体和成像生物标志物。


亨廷顿舞蹈症是一种慢性进行性神经退行性疾病，目前尚无缓解疾病的治疗方法。亨廷顿舞蹈症的已知遗传原因使得识别注定会患这种疾病的个体并在症状出现之前开始治疗成为可能。针对 HD 病因的多项试验已经在进行中，但临床测量通常对典型临床试验期间的变化不敏感。需要药物靶点参与、疾病严重程度和进展的强大生物标志物来评估治疗效果，并且正在共同努力以实现这一目标。生物流体生物标志物具有在分子水平上直接量化生物过程的潜在优势，而成像生物标志物可以在大脑结构水平上量化相关变化。最强大的生物流体和成像生物标志物可以提供补充信息，对疾病阶段和进展进行更全面的评估，为临床试验设计和终点提供信息。