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.

自从鉴定出与亨廷顿病（HD）相关的基因以来，已有25年的历史见证了有关该疾病的性质及其发病机理的深刻发现。尽管取得了这一进展，但迄今为止，疾病改善疗法的发展一直很缓慢。临床上对HD途径中断的治疗潜力的临床验证证实了新型和重新用途的药理学试剂在临床评估中的发展。最有前途的治疗方法包括降低亨廷顿蛋白（HTT）和修饰以及调节神经炎症和突触传递。随着针对这些方法中的许多方法的临床试验迫在眉睫或正在进行中，未来几年不仅对HD而言，而且对于更普遍的神经退行性疾病都有希望，