Related Experiment Video

Updated: Jan 15, 2026

Investigating the Pathogenesis of MYH7 Mutation Gly823Glu in Familial Hypertrophic Cardiomyopathy using a Mouse Model
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Investigating the Pathogenesis of MYH7 Mutation Gly823Glu in Familial Hypertrophic Cardiomyopathy using a Mouse Model

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Cardiac Myosin Inhibitors in Hypertrophic Cardiomyopathy: From Sarcomere to Clinic

Kazufumi Nakamura1,2, Takahiro Okumura3,4, Seiya Kato5

  • 1Department of Cardiovascular Medicine, Okayama University Graduate School of Medicine, Dentistry and Pharmaceutical Sciences, Okayama 700-8558, Japan.

International Journal of Molecular Sciences
|October 16, 2025
PubMed

Insights

Hypertrophic cardiomyopathy (HCM) treatments are evolving beyond symptom management. New cardiac myosin inhibitors target the sarcomere

Area of Science:

  • Cardiology
  • Molecular Biology
  • Genetics

Background:

  • Hypertrophic cardiomyopathy (HCM) is a genetic heart muscle disease causing left ventricular hypertrophy.
  • Current treatments manage symptoms but don't address underlying sarcomere hypercontractility.
  • Pathogenic variants in sarcomeric protein genes are common causes of HCM.

Purpose of the Study:

  • To review HCM pathophysiology, diagnostics, and treatments.
  • To focus on cardiac myosin inhibitors' mechanisms and clinical evidence.
  • To explore precision medicine and genetic profiling in HCM.

Main Methods:

  • Literature review of HCM pathophysiology and treatment strategies.
  • Analysis of clinical trial data for cardiac myosin inhibitors (mavacamten, aficamten).
  • Discussion of genetic profiling and precision medicine in HCM management.

Main Results:

  • Cardiac myosin inhibitors directly modulate sarcomeric function by reducing actin-myosin interactions and ATPase activity.
  • Mavacamten and aficamten show promise in improving symptoms, exercise capacity, and reducing left ventricular outflow tract gradients in obstructive HCM.
  • These inhibitors represent a shift towards targeted therapy for HCM.

Conclusions:

  • Cardiac myosin inhibitors offer a novel therapeutic approach for obstructive HCM by addressing the molecular basis of the disease.
  • Future research should focus on optimizing myosin inhibitor therapy and exploring applications in non-obstructive HCM.
  • Genetic profiling is crucial for personalized treatment strategies in HCM.

Keywords:
aficamtenheart failurehypertrophic cardiomyopathymavacamtenmyosin inhibitorssarcomere

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