Unmet needs and future directions in hypertrophic cardiomyopathy

IF 5.6 2区 医学 Q1 CARDIAC & CARDIOVASCULAR SYSTEMS
Simrat Kaur, Milind Desai
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引用次数: 0

Abstract

Hypertrophic cardiomyopathy (HCM) is a highly treatable monogenetic disorder affecting nearly 0.2% of the population. The high burden of this disease demands suitable measures for early diagnosis and preventing as well as tackling misdiagnosis. While conventionally available therapies have been efficacious in reducing symptoms, they have not been able to change the natural history of the disease. The landscape of medical treatment is rapidly changing with advent of novel pharmacotherapies such as cardiac myosin inhibitors. Ongoing investigations in gene editing have demonstrated benefits in correcting underlying genetic mutations and this is where the future of treatment lies. Contemporary procedural techniques as alternatives to available septal reduction therapies independent of coronary vascular anatomy are also emerging. This review details the recent developments, unmet needs and future directions in diagnosis, medical and invasive treatment of HCM.

肥厚型心肌病尚未满足的需求和未来发展方向。
肥厚型心肌病(HCM)是一种高度可治疗的单基因疾病,影响近0.2%的人群。这种疾病的高负担要求采取适当的措施进行早期诊断、预防和解决误诊问题。虽然传统的治疗方法在减轻症状方面是有效的,但它们并不能改变疾病的自然史。随着新型药物疗法(如心肌肌球蛋白抑制剂)的出现,医疗领域正在迅速发生变化。正在进行的基因编辑研究已经证明了纠正潜在基因突变的好处,这就是治疗的未来所在。现代手术技术作为独立于冠状血管解剖结构的可用间隔缩小疗法的替代品也在出现。这篇综述详细介绍了HCM的诊断、医疗和侵入性治疗的最新进展、未满足的需求和未来方向。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
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来源期刊
Progress in cardiovascular diseases
Progress in cardiovascular diseases 医学-心血管系统
CiteScore
10.90
自引率
6.60%
发文量
98
审稿时长
7 days
期刊介绍: Progress in Cardiovascular Diseases provides comprehensive coverage of a single topic related to heart and circulatory disorders in each issue. Some issues include special articles, definitive reviews that capture the state of the art in the management of particular clinical problems in cardiology.
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