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Hypertrophic Cardiomyopathy versus Storage Diseases with Myocardial Involvement

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MDPI
DOI: 10.3390/ijms241713239

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hypertrophic cardiomyopathy; Fabry disease; Pompe disease; Danon disease; genetics; gene therapies

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This article discusses the main cause of heart failure, which is cardiomyopathies. Among them, the most common is hypertrophic cardiomyopathy (HCM). The article also describes other cardiomyopathies with myocardial hypertrophy, including Fabry disease, Pompe disease, and Danon disease. It provides information on the genetics and pathogenesis of these diseases, as well as current and experimental treatment options such as pharmacological intervention and gene therapies. Further research is needed to evaluate the efficacy and safety of genetic approaches in treating these diseases. The article provides current knowledge and advances in the treatment of cardiomyopathies.
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.

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