Preventative therapeutic approaches for hypertrophic cardiomyopathy

Solomon, Tanya and Filipovska, Aleksandra and Hool, Livia and Viola, Helena (2021) Preventative therapeutic approaches for hypertrophic cardiomyopathy. The Journal of Physiology, 599 (14). pp.3495-3512. ISSN 0022-3751

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Link to published document: http://doi.org/10.1113/JP279410

Abstract

Sarcomeric gene mutations are associated with the development of hypertrophic cardiomyopathy (HCM). Current drug therapeutics for HCM patients are effective in relieving symptoms, but do not prevent or reverse disease progression. Moreover, due to heterogeneity in the clinical manifestations of the disease, patients experience variable outcomes in response to therapeutics. Mechanistically, alterations in calcium handling, sarcomeric disorganization, energy metabolism and contractility participate in HCM disease progression. While some similarities exist, each mutation appears to lead to mutation-specific pathophysiology. Furthermore, these alterations may precede or proceed development of the pathology. This review assesses the efficacy of HCM therapeutics from studies performed in animal models of HCM and human clinical trials. Evidence suggests that a preventative rather than corrective therapeutic approach may be more efficacious in the treatment of HCM. In addition, a clear understanding of mutation-specific mechanisms may assist in informing the most effective therapeutic mode of action.

Item Type: Article
Subjects: R Medicine > R Medicine (General)
Depositing User: Repository Administrator
Date Deposited: 30 Sep 2021 02:57
Last Modified: 30 Sep 2021 02:57
URI: http://eprints.victorchang.edu.au/id/eprint/1130

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