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马伐卡坦:肥厚型心肌病新型治疗方法综述

Mavacamten: A Review of a Novel Therapeutic Approach for Hypertrophic Cardiomyopathy.

作者信息

Abdul Qadir Memon Ayesha, Shamim Areeba, Mirza Sanoober, Osama Muhammad, Naeem Muhammad Iyad, R Wei Calvin

机构信息

Department of Pharmacy Practice, Faculty of Pharmacy, University of Karachi, Pakistan.

Department of Pharmacology, Faculty of Pharmacy, University of Karachi, Pakistan.

出版信息

Cardiovasc Hematol Agents Med Chem. 2024 Apr 5. doi: 10.2174/0118715257283752240325082733.

DOI:10.2174/0118715257283752240325082733
PMID:38584533
Abstract

Hypertrophic Cardiomyopathy (HCM) is a heart disease that can cause left ventricular hypertrophy, arrhythmias, heart failure, and sudden cardiac death. Currently, pharmacological treatment is limited and ineffective. Mavacamten (CamzyosTM) is a cardiac myosin inhibitor developed as a therapeutic option to reduce myocardial contractility and restoration of myocardial function. The Food and Drug Administration (FDA) approved the use of Mavacamten in 2022 for HCM symptoms. Clinical studies have proven that Mavacamten can reduce Left Ventricular Outflow Tract (LVOT) involvement, cardiac hypercontraction, and hypertrophy. This review provides an overview of HCM, its pathophysiology, current treatments, synthesis of Mavacamten, and the clinical trials of Mavacamten.

摘要

肥厚型心肌病(HCM)是一种可导致左心室肥厚、心律失常、心力衰竭和心源性猝死的心脏病。目前,药物治疗有限且效果不佳。马伐卡坦(CamzyosTM)是一种开发用于降低心肌收缩力和恢复心肌功能的心肌肌球蛋白抑制剂。美国食品药品监督管理局(FDA)于2022年批准将马伐卡坦用于治疗HCM症状。临床研究证明,马伐卡坦可减少左心室流出道(LVOT)受累、心脏过度收缩和肥厚。本综述概述了HCM、其病理生理学、当前治疗方法、马伐卡坦的合成以及马伐卡坦的临床试验。

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