Overview
Dilated cardiomyopathy (DCM), also known as congestive cardiomyopathy, is a heart muscle disease characterized by enlargement (dilation) and impaired contraction of the left ventricle or both ventricles. The heart chambers stretch and thin, reducing the heart's ability to pump blood efficiently throughout the body. This progressive condition primarily affects the cardiovascular system and can lead to heart failure, arrhythmias (abnormal heart rhythms), thromboembolism (blood clots), and sudden cardiac death. DCM can be caused by genetic mutations (familial DCM, accounting for 20–50% of cases), or it may be acquired through viral infections, alcohol use, toxins, autoimmune conditions, or other systemic diseases. In many cases, no identifiable cause is found (idiopathic DCM). Key symptoms include shortness of breath (dyspnea), particularly during exertion or when lying flat, fatigue, exercise intolerance, peripheral edema (swelling of the legs and ankles), palpitations, and in advanced cases, signs of congestive heart failure such as fluid retention and reduced exercise capacity. Some patients may be asymptomatic in early stages, with the condition discovered incidentally through imaging studies. Arrhythmias, including atrial fibrillation and ventricular tachycardia, are common complications that can significantly impact prognosis. Treatment of dilated cardiomyopathy focuses on managing heart failure symptoms and preventing complications. Standard medical therapy includes ACE inhibitors or angiotensin receptor blockers, beta-blockers, mineralocorticoid receptor antagonists, and diuretics. Newer agents such as sacubitril/valsartan (ARNI) and SGLT2 inhibitors have also become part of guideline-directed medical therapy. Anticoagulation may be prescribed to reduce thromboembolic risk. Implantable cardioverter-defibrillators (ICDs) are recommended for patients at high risk of sudden cardiac death, and cardiac resynchronization therapy (CRT) may benefit those with conduction abnormalities. In end-stage disease, left ventricular assist devices (LVADs) or heart transplantation may be considered. Familial cases warrant genetic counseling and screening of first-degree relatives.
Variable
Can be inherited in different ways depending on the underlying gene
Variable
Can begin at different ages, from infancy through adulthood
FDA & Trial Timeline
10 eventsRocket Pharmaceuticals Inc. — PHASE1
Assiut University
Fondazione Policlinico Universitario Agostino Gemelli IRCCS
Affinia Therapeutics — PHASE1, PHASE2
Thomas Roston — NA
Second Affiliated Hospital, School of Medicine, Zhejiang University
University Hospital, Essen — NA
Sebastiano Lava — PHASE2
Data sourced from FDA regulatory filings and ClinicalTrials.gov. Updated periodically.
Treatments
No FDA-approved treatments are currently listed for Dilated cardiomyopathy.
20 clinical trialsare actively recruiting — trials can provide access to cutting-edge therapies.
View clinical trials →Rare Disease Specialist
Treatment Centers
8 centersBaylor College of Medicine Rare Disease Center ↗
Baylor College of Medicine
📍 Houston, TX
🏥 NORDStanford Medicine Rare Disease Center ↗
Stanford Medicine
📍 Stanford, CA
🔬 UDNNIH Clinical Center Undiagnosed Diseases Program ↗
National Institutes of Health
📍 Bethesda, MD
🔬 UDNUCLA UDN Clinical Site ↗
UCLA Health
📍 Los Angeles, CA
🔬 UDNBaylor College of Medicine UDN Clinical Site ↗
Baylor College of Medicine
📍 Houston, TX
🔬 UDNHarvard/MGH UDN Clinical Site ↗
Massachusetts General Hospital
📍 Boston, MA
🏥 NORDMayo Clinic Center for Individualized Medicine ↗
Mayo Clinic
📍 Rochester, MN
👤 Mayo Clinic Center for Individualized Medicine
🏥 NORDUCLA Rare Disease Day Program ↗
UCLA Health
📍 Los Angeles, CA
Travel Grants
No travel grants are currently matched to Dilated cardiomyopathy.
Community
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Common questions about Dilated cardiomyopathy
What is Dilated cardiomyopathy?
Dilated cardiomyopathy (DCM), also known as congestive cardiomyopathy, is a heart muscle disease characterized by enlargement (dilation) and impaired contraction of the left ventricle or both ventricles. The heart chambers stretch and thin, reducing the heart's ability to pump blood efficiently throughout the body. This progressive condition primarily affects the cardiovascular system and can lead to heart failure, arrhythmias (abnormal heart rhythms), thromboembolism (blood clots), and sudden cardiac death. DCM can be caused by genetic mutations (familial DCM, accounting for 20–50% of cases),
Are there clinical trials for Dilated cardiomyopathy?
Yes — 20 recruiting clinical trials are currently listed for Dilated cardiomyopathy on UniteRare. See the clinical trials section on this page for phase, sponsor, and site details sourced from ClinicalTrials.gov.
Which specialists treat Dilated cardiomyopathy?
25 specialists and care centers treating Dilated cardiomyopathy are listed on UniteRare, sourced from ClinicalTrials.gov principal investigators, published research, and the NPPES NPI registry.