Background Despite the monogenic autosomal dominant nature of Huntington’s disease (HD), the current research paradigm is still based on overt clinical phenotypes and does not address disease pathobiology and biomarkers that are evident decades before functional decline. A new research framework is needed to standardize clinical research and enable interventional studies earlier in the course of HD.

中文翻译：

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亨廷顿病综合分期系统 (HD-ISS)：一种新的循证分期分类系统

背景尽管亨廷顿病 (HD) 具有单基因常染色体显性遗传，但当前的研究范式仍基于明显的临床表型，并未解决在功能衰退前几十年就已明显的疾病病理生物学和生物标志物。需要一个新的研究框架来规范临床研究，并在 HD 过程的早期实现介入研究。