探索 Deutetrabenazine 在治疗与亨廷顿舞蹈症相关的舞蹈症中的作用。

Siddhant Tripathi, Yashika Sharma, Dileep Kumar
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引用次数: 0

摘要

背景:这篇综述探讨了去甲替拉嗪治疗与 HD 相关的舞蹈症的疗效。运动、心理和认知症状是 HD 这种神经退行性疾病的特征。舞蹈症是与 HD 相关的一种常见运动障碍,会导致肌肉出现无法控制的抽搐动作。目前尚无治愈 HD 的方法,主要目标是通过各种药物治疗来控制症状。由于舞蹈症对患者的生活质量有很大影响,因此有效的治疗至关重要。Dutetrabenazine是首个获得美国食品药品管理局(FDA)批准用于治疗亨廷顿氏病(HD)舞蹈症的氘代药物:治疗与亨廷顿舞蹈症相关的舞蹈症可能会从使用去乙酰丙嗪中获益。新型化合物 deutetrabenazine 含有氘。它能抑制 CYP2D6 代谢,延长活性代谢物的半衰期,在保持显著药理作用的同时可能导致持续的全身暴露。Deutetrabenazine 通过抑制 VMAT2 囊泡单胺转运体,减少突触间隙中单胺(包括多巴胺)的释放。这种机制对舞蹈症有治疗作用。为治疗HD患者的舞蹈症和迟发性运动障碍,FDA于2017年批准了去甲替拉嗪(deutetrabenazine).结论:在此,我们强调,去甲替拉嗪是治疗亨廷顿氏病舞蹈症的一种很有前景的新疗法,对于舞蹈症患者来说,去甲替拉嗪为提高生活质量带来了希望。由于研究结果喜忧参半,要完全了解其有效性和潜在优势,还需要进行更多的研究,包括直接比较研究。
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Exploring the Role of Deutetrabenazine in the Treatment of Chorea Linked with Huntington's Disease.

Background: This review investigates the efficacy of deutetrabenazine in the management of chorea related to HD. Motor, psychological, and cognitive symptoms characterize HD, a neurodegenerative disease. One prominent movement disorder associated with HD is chorea, which results in uncontrollably jerky movements of the muscles. HD has no known cure; instead, symptom management with a variety of medication options is the main goal. Effective management is essential because chorea has a significant impact on patients' quality of life. Dutetrabenazine is the first deuterated medication to receive approval from the US Food and Drug Administration (FDA) for the therapeutic treatment of chorea in Huntington's disease (HD).

Objectives: Treating chorea associated with HD may benefit from the use of deutetrabenazine. The novel compound deutetrabenazine contains deuterium. It inhibits CYP2D6 metabolism, prolongs the half-lives of active metabolites, and may cause persistent systemic exposure while maintaining significant pharmacological action. Deutetrabenazine decreases the release of monoamines, including dopamine, in the synaptic cleft by inhibiting the VMAT2 vesicular monoamine transporter. For chorea, this mechanism has a therapeutic effect. For the treatment of choreiform movement and tardive dyskinesia in HD, the FDA approved deutetrabenazine in 2017.

Conclusion: Here we highlight, Deutetrabenazine as a promising new treatment for Huntington's disease chorea, for patients with chorea, deutetrabenazine offers hope for an enhanced quality of life. To completely understand its effectiveness and potential advantages, additional research is necessary, including direct comparison studies, as a result of the mixed study results.

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来源期刊
CiteScore
4.80
自引率
9.10%
发文量
55
期刊最新文献
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