January 29, 2015Open Access
View Full PaperTargets for therapy in sarcomeric cardiomyopathies
Key Result
Novel therapies targeting genetic defects, altered sarcomere function, ion homeostasis, and myocardial energetics may prevent, delay, or reverse hypertrophic cardiomyopathy.
Structured PICO
P
Population
Individuals with sarcomeric cardiomyopathies (hypertrophic cardiomyopathy caused by sarcomeric gene mutations)
I
Intervention
Novel therapies targeting genetic defects, altered sarcomere function, intracellular ion homeostasis, and impaired myocardial energetics
This review highlights emerging molecular targets that could lead to disease-modifying therapies for sarcomeric cardiomyopathies.
Abstract
To date, no compounds or interventions exist that treat or prevent sarcomeric cardiomyopathies. Established therapies currently improve the outcome, but novel therapies may be able to more fundamentally affect the disease process and course. Investigations of the pathomechanisms are generating molecular insights that can be useful for the design of novel specific drugs suitable for clinical use. As perturbations in the heart are stage-specific, proper timing of drug treatment is essential to prevent initiation and progression of cardiac disease in mutation carrier individuals. In this review, we emphasize potential novel therapies which may prevent, delay, or even reverse hypertrophic cardiomyopathy caused by sarcomeric gene mutations. These include corrections of genetic defects, altered sarcomere function, perturbations in intracellular ion homeostasis, and impaired myocardial energetics.
Cite This Study
Tardiff et al. (Thu,) conducted a review in Sarcomeric cardiomyopathies. Novel therapies targeting pathomechanisms was evaluated. Novel therapies targeting genetic defects, altered sarcomere function, ion homeostasis, and myocardial energetics may prevent, delay, or reverse hypertrophic cardiomyopathy.