First Department of Cardiology, Medical University of Warsaw, ul. Banacha 1A, 02-097 Warszawa, Poland.
Doctoral School, Medical University of Warsaw, 81 Żwirki i Wigury Street, 02-091 Warsaw, Poland.
Int J Mol Sci. 2023 Aug 26;24(17):13239. doi: 10.3390/ijms241713239.
One of the main causes of heart failure is cardiomyopathies. Among them, the most common is hypertrophic cardiomyopathy (HCM), characterized by thickening of the left ventricular muscle. This article focuses on HCM and other cardiomyopathies with myocardial hypertrophy, including Fabry disease, Pompe disease, and Danon disease. The genetics and pathogenesis of these diseases are described, as well as current and experimental treatment options, such as pharmacological intervention and the potential of gene therapies. Although genetic approaches are promising and have the potential to become the best treatments for these diseases, further research is needed to evaluate their efficacy and safety. This article describes current knowledge and advances in the treatment of the aforementioned cardiomyopathies.
心力衰竭的主要原因之一是心肌病。其中，最常见的是肥厚型心肌病（HCM），其特征是左心室肌肉增厚。本文重点介绍 HCM 和其他伴有心肌肥厚的心肌病，包括法布瑞氏病、庞贝病和丹顿病。描述了这些疾病的遗传学和发病机制，以及当前和实验性的治疗选择，如药物干预和基因治疗的潜力。尽管遗传方法很有前景，并有潜力成为这些疾病的最佳治疗方法，但仍需要进一步研究来评估其疗效和安全性。本文描述了上述心肌病的治疗的现有知识和进展。
