中国实用神经疾病杂志2026，Vol.29Issue(3)：373-379,7.DOI:10.12083/SYSJ.251062
VMAT2抑制剂治疗亨廷顿舞蹈病的研究进展
Therapeutic advances of VMAT2 inhibitors in Huntington's disease
摘要
Abstract
Huntington's disease(HD)is an autosomal dominant neurodegenerative disease,characterized clinically by choreiform involuntary movements,cognitive impairment and psychiatric symptoms,with insidious onset,slow progression,poor prognosis,high rate of disability,and a heavy disease burden.At present,no mature disease-modifying therapy is available,and pharmacological management remains largely symptomatic.Vesicular monoamine transporter 2(VMAT2)inhibitors reduce excessive dopaminergic overactivity in the striatum by inhibiting presynaptic vesicular monoamine uptake and depleting dopamine stores,and have become one of the main drug options for the treatment of chorea in patients with HD,including tetrabenazine,deutetrabenazine and valbenazine.This review summarizes the pharmacological properties,clinical evidence and safety profiles of these VMAT2 inhibitors.Current studies indicate that all three VMAT2 inhibitors can significantly improve choreic symptoms in patients with HD.However,adverse effects such as depression,increased suicide risk and extrapyramidal symptoms still warrant close attention.Future head-to-head trials and real-world studies are needed to further optimize patient selection and dosing strategies for different agents and to explore their integration with potential disease-modifying therapies.关键词
亨廷顿舞蹈病/舞蹈症/囊泡单胺转运体2抑制剂/药物治疗Key words
Huntington's disease/Chorea/Vesicular monoamine transporter 2 inhibitors/Drug therapy分类
医药卫生引用本文复制引用
吴嫣云,欧汝威..VMAT2抑制剂治疗亨廷顿舞蹈病的研究进展[J].中国实用神经疾病杂志,2026,29(3):373-379,7.基金项目
国家自然科学基金资助项目(编号:82271272) （编号:82271272）
