Non-familial rare disease with dilated cardiomyopathy

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1Active trials8Treatment centers

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Overview

Non-familial dilated cardiomyopathy is a rare heart condition where the heart's main pumping chamber (the left ventricle) becomes enlarged and weakened. Unlike familial forms of dilated cardiomyopathy, this type does not run in families and is not caused by an inherited gene mutation passed from parent to child. Instead, it may arise from other causes such as viral infections, autoimmune reactions, toxic exposures (like alcohol or certain medications), or sometimes the cause remains unknown (idiopathic). When the heart muscle stretches and thins, it cannot pump blood as effectively as it should, leading to heart failure symptoms. Key symptoms include shortness of breath (especially during activity or when lying down), fatigue, swelling in the legs and ankles, irregular heartbeats, and dizziness. Some people may also experience chest discomfort or fainting spells. The severity can range from mild to life-threatening. Treatment focuses on managing heart failure and improving heart function. This typically includes medications such as ACE inhibitors, beta-blockers, and diuretics. In more advanced cases, devices like implantable cardioverter-defibrillators (ICDs) or cardiac resynchronization therapy (CRT) may be needed. Heart transplantation may be considered for the most severe cases that do not respond to other treatments. Early diagnosis and consistent medical follow-up are important for improving outcomes.

Key symptoms:

Shortness of breath, especially with activityFatigue and low energySwelling in the legs, ankles, or feetIrregular or rapid heartbeatDizziness or lightheadednessDifficulty breathing when lying flatChest discomfort or pressureFainting or near-fainting episodesReduced ability to exercisePersistent cough or wheezingSudden weight gain from fluid retentionLoss of appetite

Inheritance

Sporadic

Usually appears on its own, not inherited from a parent

Age of Onset

Variable

Can begin at different ages, from infancy through adulthood

Orphanet ↗NORD ↗

FDA & Trial Timeline

1 event
Jun 2016DCM Precision Medicine Study

Ray Hershberger — NA

TrialACTIVE NOT RECRUITING

Data sourced from FDA regulatory filings and ClinicalTrials.gov. Updated periodically.

Treatments

No FDA-approved treatments are currently listed for Non-familial rare disease with dilated cardiomyopathy.

1 clinical trialare actively recruiting — trials can provide access to cutting-edge therapies.

View clinical trials →

Clinical Trials

1 recruitingView all trials with filters →
N/A1 trial
DCM Precision Medicine Study
N/A
Active
PI: Ray Hershberger, MD (Ohio State University) · Sites: Birmingham, Alabama; Tucson, Arizona +24 more

No specialists are currently listed for Non-familial rare disease with dilated cardiomyopathy.

View NORD Rare Disease Centers ↗Undiagnosed Disease Network ↗

Treatment Centers

8 centers
🏥 NORD

Baylor College of Medicine Rare Disease Center

Baylor College of Medicine

📍 Houston, TX

🏥 NORD

Stanford Medicine Rare Disease Center

Stanford Medicine

📍 Stanford, CA

🔬 UDN

NIH Clinical Center Undiagnosed Diseases Program

National Institutes of Health

📍 Bethesda, MD

🔬 UDN

UCLA UDN Clinical Site

UCLA Health

📍 Los Angeles, CA

🔬 UDN

Baylor College of Medicine UDN Clinical Site

Baylor College of Medicine

📍 Houston, TX

🔬 UDN

Harvard/MGH UDN Clinical Site

Massachusetts General Hospital

📍 Boston, MA

🏥 NORD

Mayo Clinic Center for Individualized Medicine

Mayo Clinic

📍 Rochester, MN

👤 Mayo Clinic Center for Individualized Medicine

🏥 NORD

UCLA Rare Disease Day Program

UCLA Health

📍 Los Angeles, CA

Travel Grants

No travel grants are currently matched to Non-familial rare disease with dilated cardiomyopathy.

Search all travel grants →NORD Financial Assistance ↗

Community

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Caregiver Resources

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Social Security Disability

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Questions for your doctor

Bring these to your next appointment

  • Q1.What is the likely cause of my dilated cardiomyopathy, and can it be treated or reversed?,What is my current heart function (ejection fraction), and what does that number mean for me?,Which medications do I need, and what side effects should I watch for?,Do I need an implantable device like an ICD or CRT, and why?,What activity level is safe for me, and should I participate in cardiac rehabilitation?,Should my family members be screened for heart problems even though this is non-familial?,What warning signs should prompt me to seek emergency care?

Common questions about Non-familial rare disease with dilated cardiomyopathy

What is Non-familial rare disease with dilated cardiomyopathy?

Non-familial dilated cardiomyopathy is a rare heart condition where the heart's main pumping chamber (the left ventricle) becomes enlarged and weakened. Unlike familial forms of dilated cardiomyopathy, this type does not run in families and is not caused by an inherited gene mutation passed from parent to child. Instead, it may arise from other causes such as viral infections, autoimmune reactions, toxic exposures (like alcohol or certain medications), or sometimes the cause remains unknown (idiopathic). When the heart muscle stretches and thins, it cannot pump blood as effectively as it shoul

How is Non-familial rare disease with dilated cardiomyopathy inherited?

Non-familial rare disease with dilated cardiomyopathy follows a sporadic inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.

Are there clinical trials for Non-familial rare disease with dilated cardiomyopathy?

Yes — 1 recruiting clinical trial is currently listed for Non-familial rare disease with dilated cardiomyopathy on UniteRare. See the clinical trials section on this page for phase, sponsor, and site details sourced from ClinicalTrials.gov.