Preventative therapeutic approaches for hypertrophic cardiomyopathy

Research output: Contribution to journalReview articlepeer-review

6 Citations (Scopus)

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. (Figure presented.).

Original languageEnglish
Pages (from-to)3495-3512
Number of pages18
JournalJournal of Physiology
Volume599
Issue number14
Early online date21 Aug 2020
DOIs
Publication statusPublished - 15 Jul 2021

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