Intermediate epidermolysis bullosa simplex with cardiomyopathy

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ORPHA:508529OMIM:617294Q81.0
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Overview

Intermediate epidermolysis bullosa simplex with cardiomyopathy (also sometimes called EBS with cardiomyopathy or EBS-MD-MyS in older literature) is a very rare inherited skin and heart condition. 'Epidermolysis bullosa simplex' means the skin blisters easily because the cells that hold the outer layer of skin together are fragile. The 'intermediate' part describes how severe the blistering is — more than mild forms but not as severe as the most serious types. The 'cardiomyopathy' part means the heart muscle is also affected, becoming weakened or thickened over time. People with this condition typically develop painful blisters on the skin from minor friction or bumps, often starting at birth or in early infancy. The blisters usually appear on the hands, feet, and other areas that experience rubbing. Over time, heart problems develop, which can include a weakened or enlarged heart muscle that does not pump blood as well as it should. There is currently no cure for this condition. Treatment focuses on protecting the skin from injury, carefully managing wounds and blisters, and monitoring and treating the heart with medications used for heart failure or cardiomyopathy. A team of specialists is needed to manage both the skin and heart aspects of this disease throughout a person's life.

Also known as:

Key symptoms:

Skin blisters that form easily from minor rubbing or bumpingBlisters on the hands and feet, especially the palms and solesPainful, slow-healing skin woundsThickened or hardened skin on the palms and soles over timeWeakened heart muscle (dilated or hypertrophic cardiomyopathy)Shortness of breath due to heart problemsFatigue and low energy, especially with activityIrregular heartbeat (arrhythmia) in some casesSwelling in the legs or feet from heart failureNail changes or loss of nailsScarring of the skin after blisters heal

Inheritance

Autosomal recessive

Passed on when both parents carry the same gene change; often skips generations

Age of Onset

Neonatal

Begins at or shortly after birth (first 4 weeks)

Orphanet ↗OMIM ↗NORD ↗

Treatments

No FDA-approved treatments are currently listed for Intermediate epidermolysis bullosa simplex with cardiomyopathy.

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No actively recruiting trials found for Intermediate epidermolysis bullosa simplex with cardiomyopathy at this time.

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No specialists are currently listed for Intermediate epidermolysis bullosa simplex with 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 Intermediate epidermolysis bullosa simplex with cardiomyopathy.

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Community

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Caregiver Resources

NORD Caregiver Resources

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Mental Health Support

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Social Security Disability

Learn how rare disease patients may qualify for SSDI/SSI benefits.

Questions for your doctor

Bring these to your next appointment

  • Q1.Which specific gene change has been found in my case, and what does that mean for my prognosis?,How often should my heart be checked, and what signs should prompt me to seek urgent care?,What wound care routine do you recommend, and can I be referred to a specialist EB nurse?,Are there any clinical trials or research studies I might be eligible for?,Should my family members be tested for this condition?,What heart medications are appropriate for me, and what are the side effects I should watch for?,Are there any EB specialist centers or multidisciplinary clinics you can refer me to?

Common questions about Intermediate epidermolysis bullosa simplex with cardiomyopathy

What is Intermediate epidermolysis bullosa simplex with cardiomyopathy?

Intermediate epidermolysis bullosa simplex with cardiomyopathy (also sometimes called EBS with cardiomyopathy or EBS-MD-MyS in older literature) is a very rare inherited skin and heart condition. 'Epidermolysis bullosa simplex' means the skin blisters easily because the cells that hold the outer layer of skin together are fragile. The 'intermediate' part describes how severe the blistering is — more than mild forms but not as severe as the most serious types. The 'cardiomyopathy' part means the heart muscle is also affected, becoming weakened or thickened over time. People with this condition

How is Intermediate epidermolysis bullosa simplex with cardiomyopathy inherited?

Intermediate epidermolysis bullosa simplex with cardiomyopathy follows a autosomal recessive inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.

At what age does Intermediate epidermolysis bullosa simplex with cardiomyopathy typically begin?

Typical onset of Intermediate epidermolysis bullosa simplex with cardiomyopathy is neonatal. Age of onset can vary across affected individuals.