Cargando…
Preventative therapeutic approaches for hypertrophic cardiomyopathy
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...
Autores principales: | , , , |
---|---|
Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
John Wiley and Sons Inc.
2020
|
Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8359240/ https://www.ncbi.nlm.nih.gov/pubmed/32822065 http://dx.doi.org/10.1113/JP279410 |
_version_ | 1783737504799653888 |
---|---|
author | Solomon, Tanya Filipovska, Aleksandra Hool, Livia Viola, Helena |
author_facet | Solomon, Tanya Filipovska, Aleksandra Hool, Livia Viola, Helena |
author_sort | Solomon, Tanya |
collection | PubMed |
description | 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. [Image: see text] |
format | Online Article Text |
id | pubmed-8359240 |
institution | National Center for Biotechnology Information |
language | English |
publishDate | 2020 |
publisher | John Wiley and Sons Inc. |
record_format | MEDLINE/PubMed |
spelling | pubmed-83592402021-08-17 Preventative therapeutic approaches for hypertrophic cardiomyopathy Solomon, Tanya Filipovska, Aleksandra Hool, Livia Viola, Helena J Physiol Symposium section reviews: unravelling the mysteries of mitochondria in health and disease 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. [Image: see text] John Wiley and Sons Inc. 2020-09-07 2021-07-15 /pmc/articles/PMC8359240/ /pubmed/32822065 http://dx.doi.org/10.1113/JP279410 Text en © 2020 The Authors. The Journal of Physiology published by John Wiley & Sons Ltd on behalf of The Physiological Society https://creativecommons.org/licenses/by/4.0/This is an open access article under the terms of the http://creativecommons.org/licenses/by/4.0/ (https://creativecommons.org/licenses/by/4.0/) License, which permits use, distribution and reproduction in any medium, provided the original work is properly cited. |
spellingShingle | Symposium section reviews: unravelling the mysteries of mitochondria in health and disease Solomon, Tanya Filipovska, Aleksandra Hool, Livia Viola, Helena Preventative therapeutic approaches for hypertrophic cardiomyopathy |
title | Preventative therapeutic approaches for hypertrophic cardiomyopathy |
title_full | Preventative therapeutic approaches for hypertrophic cardiomyopathy |
title_fullStr | Preventative therapeutic approaches for hypertrophic cardiomyopathy |
title_full_unstemmed | Preventative therapeutic approaches for hypertrophic cardiomyopathy |
title_short | Preventative therapeutic approaches for hypertrophic cardiomyopathy |
title_sort | preventative therapeutic approaches for hypertrophic cardiomyopathy |
topic | Symposium section reviews: unravelling the mysteries of mitochondria in health and disease |
url | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8359240/ https://www.ncbi.nlm.nih.gov/pubmed/32822065 http://dx.doi.org/10.1113/JP279410 |
work_keys_str_mv | AT solomontanya preventativetherapeuticapproachesforhypertrophiccardiomyopathy AT filipovskaaleksandra preventativetherapeuticapproachesforhypertrophiccardiomyopathy AT hoollivia preventativetherapeuticapproachesforhypertrophiccardiomyopathy AT violahelena preventativetherapeuticapproachesforhypertrophiccardiomyopathy |