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Updated: Dec 31, 2025

Investigating the Pathogenesis of MYH7 Mutation Gly823Glu in Familial Hypertrophic Cardiomyopathy using a Mouse Model
Published on: August 8, 2022
Pharmacotherapy for the treatment of obstructive hypertrophic cardiomyopathy
R Spoladore1,2, G Fragasso2,3, L Pannone2
1Head - Referral ambulatory for Hypertrophy Cardiomyopathy, IRCCS San Raffaele University Hospital, Milan, Italy.
Insights
Hypertrophic cardiomyopathy (HCM) treatment is evolving. Current research focuses on symptom control, metabolic modulation, and novel genetic therapies for this common genetic heart disease.
Area of Science:
- Cardiology
- Genetics
- Pharmacology
Background:
- Hypertrophic cardiomyopathy (HCM) is a prevalent genetic heart disease.
- It is a leading cause of sudden cardiac death, heart failure, and stroke.
- Left ventricular outflow tract (LVOT) obstruction is a key feature of HCM.
Purpose of the Study:
- To review recent advancements in the pharmacological treatment of obstructive HCM.
- To explore interventions targeting symptom control, cardiac metabolism, and disease modification.
- To discuss potential genetic preventive therapies.
Main Methods:
- Review of recent scientific literature on obstructive HCM pharmacotherapy.
- Analysis of interventions including symptom management, metabolic modulation, and genetic therapies.
- Synthesis of expert opinions on current and future treatment strategies.
Main Results:
- Limited data currently support pharmacological interventions for HCM.
- Novel treatment options are emerging with a better understanding of HCM pathophysiology.
- Further research is needed on drugs with previously mixed results and larger trials for promising molecules.
Conclusions:
- Improved understanding of HCM pathophysiology is crucial for developing new treatments.
- Future research should focus on re-evaluating drugs with limited past efficacy.
- Larger clinical trials are essential to validate positive findings for pressure gradient and symptom control.
Abstract:
Introduction: Hypertrophic cardiomyopathy (HCM) is one of the most common genetic heart diseases and represents a leading cause of sudden cardiac death as well as a prevalent cause of heart failure and stroke. HCM is characterized by a very complex pathophysiology, consisting of heterogeneous clinical manifestations and natural history. Left ventricular outflow tract (LVOT) obstruction has been considered the most knowable feature of HCM since the initial clinical descriptions of the disease.Areas covered: In this review, the authors discuss the most recent reports on the pharmacological treatment of obstructive HCM, mainly based on three different levels of intervention: control of symptoms, cardiac metabolism modulation and disease-modifying approaches, including genetic preventive therapies.Expert opinion: There are presently limited data supporting pharmacological interventions for this complex disease. However, an improved understanding of HCM pathophysiology will allow the development of novel treatment options. Two important key messages are to further study drugs with negative but limited previous results and to design new and larger trials for those molecules that have already produced positive results in HCM, especially for pressure gradients and symptoms control.
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