A Narrative Review of Current and Investigational Therapies in Hypertrophic Cardiomyopathy

Ian Ogurek1, Randeep Gill1, Vasiliki Tasouli-Drakou1

  • 1Department of Internal Medicine, Kirk Kerkorian School of Medicine, University of Nevada, Las Vegas, NV 89106, USA.

Biomedicines
|June 26, 2025
PubMed

Insights

Hypertrophic cardiomyopathy, an inherited heart condition, is seeing new treatments like cardiac myosin inhibitors and gene editing. This review covers current and future therapies for improved patient management.

Area of Science:

  • Cardiology
  • Genetics
  • Pharmacology

Background:

  • Hypertrophic cardiomyopathy (HCM) is the most common inherited cardiac disease.
  • It follows an autosomal dominant inheritance pattern.
  • Diagnosis relies on imaging (echocardiography, cardiac MRI) and genetic testing.

Purpose of the Study:

  • To review current and investigational treatments for hypertrophic cardiomyopathy.
  • To highlight recent advancements in managing this prevalent inherited cardiac condition.

Main Methods:

  • Literature review of pharmacological and invasive therapies.
  • Inclusion of approved treatments such as cardiac myosin inhibitors.
  • Discussion of emerging approaches like radiofrequency ablation and gene editing.

Main Results:

  • Significant advancements in HCM management are transforming patient care.
  • New therapeutic options offer improved treatment strategies.
  • Ongoing clinical trials are exploring novel investigational therapies.

Conclusions:

  • Current and future treatments offer new hope for hypertrophic cardiomyopathy patients.
  • The landscape of HCM management is rapidly evolving.
  • Further research and clinical trials are crucial for optimizing outcomes.

Related Concept Videos

Cardiomyopathy V: Interprofessional Care01:29

Cardiomyopathy V: Interprofessional Care

Managing cardiomyopathy involves addressing underlying or precipitating causes, treating heart failure with medications, and implementing dietary changes and a balanced exercise and rest regimen.Lifestyle ModificationsCardiomyopathy patients should adopt a low-sodium diet to reduce fluid retention and manage heart failure. A personalized exercise and rest plan helps maintain physical fitness without overstraining the heart. Avoiding alcohol and tobacco is essential to prevent further damage to...
41
Cardiomyopathy III: Hypertrophic Cardiomyopathy01:29

Cardiomyopathy III: Hypertrophic Cardiomyopathy

Hypertrophic cardiomyopathy, or HCM, is an autosomal dominant genetic disorder characterized by asymmetric left ventricular hypertrophy without ventricular dilation. It is more common in men and is typically diagnosed in young, athletic adults.EtiologyHCM is primarily genetic and is caused by mutations in genes encoding sarcomeric proteins. Researchers have identified over 1400 mutations across at least 11 different genes. Among these, the most frequently occurring mutations are found in the...
65
Cardiomyopathy II: Dilated Cardiomyopathy01:30

Cardiomyopathy II: Dilated Cardiomyopathy

Dilated cardiomyopathy, or DCM, is a progressive myocardial disorder characterized by ventricular chamber dilation and contractile dysfunction.EtiologyVarious factors can cause DCM, including hypertension and heavy alcohol intake, which contribute to the weakening and enlargement of the heart muscle. Viral infections, such as Coxsackievirus B, adenoviruses, and influenza, can lead to DCM by causing inflammation and damage to heart tissue. Certain chemotherapeutic agents, including daunorubicin,...
30
Cardiomyopathy IV: Restrictive Cardiomyopathy01:29

Cardiomyopathy IV: Restrictive Cardiomyopathy

Restrictive cardiomyopathy (RCM) is a rare heart muscle disease characterized by impaired ventricular filling due to stiffened ventricular walls, leading to significant diastolic dysfunction.EtiologyRestrictive cardiomyopathy can arise from both inherited and acquired diseases, many of which are systemic. It is categorized into four main types: infiltrative, storage, non-infiltrative, and endomyocardial diseases.Infiltrative diseases, such as amyloidosis, lead to RCM by depositing amyloid...
44
Heart Failure Drugs: Inhibitors of Renin-Angiotensin System01:26

Heart Failure Drugs: Inhibitors of Renin-Angiotensin System

The activation of the sympathetic nervous system and the renin-angiotensin-aldosterone system (RAAS) contributes to cardiac remodeling, and inhibiting the RAAS is a pharmacological target in heart failure management. As a result, neurohumoral modulation is a crucial treatment principle for managing heart failure. This approach involves using medications like ACE inhibitors (ACEIs), angiotensin receptor blockers (ARBs), β-blockers, mineralocorticoid receptor antagonists (MRAs), and neutral...
524
Cardiomyopathy I: Introduction and Classification01:25

Cardiomyopathy I: Introduction and Classification

Cardiomyopathy, or CMP, is a group of diseases affecting the myocardial structure, impairing its ability to pump blood effectively. This condition can lead to arrhythmias, heart failure, or sudden cardiac death.Cardiomyopathies are classified into primary and secondary categories:Primary Cardiomyopathy refers to conditions involving only the heart muscle that are often idiopathic (of unknown cause) or genetic. They primarily affect the myocardium without the involvement of other systemic...
72