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Review
. 2023 Aug 26;24(17):13239.
doi: 10.3390/ijms241713239.

Hypertrophic Cardiomyopathy versus Storage Diseases with Myocardial Involvement

Anna Burban  1   2 , Szymon Pucyło  1 , Aleksandra Sikora  1 , Grzegorz Opolski  1 , Marcin Grabowski  1 , Agnieszka Kołodzińska  1
Affiliations
Review

Hypertrophic Cardiomyopathy versus Storage Diseases with Myocardial Involvement

Anna Burban et al. Int J Mol Sci. .

Abstract

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.

Keywords: Danon disease; Fabry disease; Pompe disease; gene therapies; genetics; hypertrophic cardiomyopathy.

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Conflict of interest statement

The authors declare that there are no conflict of interest.

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