Genetic cardiac malformation

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ORPHA:477805
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22Specialists8Treatment centers

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Overview

Genetic cardiac malformation (Orphanet code 477805) is a broad term used to describe heart defects that are caused — at least in part — by changes in a person's genes. These malformations happen when the heart does not form correctly during early development in the womb. The result can be structural problems such as holes between the heart's chambers, abnormal valves, or vessels that are connected in the wrong way. Some people also hear this group of conditions called 'congenital heart defects with a genetic cause' or 'hereditary congenital heart disease.' Because this is a category covering many different types of heart malformations, symptoms can vary widely from person to person. Some individuals have mild defects that cause few problems, while others have complex heart anatomy that requires surgery shortly after birth. Common symptoms include a bluish tint to the skin (cyanosis), rapid or difficult breathing, poor feeding in infants, fatigue, and reduced ability to exercise. Some people also have other birth differences in other parts of the body, depending on the underlying genetic cause. Treatment depends on the specific type of heart defect. Options range from careful monitoring with no immediate intervention, to medications that help the heart work more efficiently, to catheter-based procedures or open-heart surgery. Many people with genetic cardiac malformations live full and active lives, especially when the condition is caught early and managed by a specialist team. Ongoing follow-up with a cardiologist throughout life is usually recommended.

Key symptoms:

Bluish or grayish color of the skin, lips, or fingernails (cyanosis)Rapid or labored breathingPoor feeding or tiring easily during feeding in infantsUnusual fatigue or low energyReduced ability to exercise or keep up with peersHeart murmur detected by a doctorSwelling in the legs, belly, or around the eyesFainting or near-fainting spellsIrregular or fast heartbeat (palpitations)Slow weight gain or poor growth in infants and childrenChest pain or discomfortFrequent respiratory infections

Inheritance

Variable

Can be inherited in different ways depending on the underlying gene

Age of Onset

Variable

Can begin at different ages, from infancy through adulthood

Orphanet ↗NORD ↗

Treatments

No FDA-approved treatments are currently listed for Genetic cardiac malformation.

View clinical trials →

No actively recruiting trials found for Genetic cardiac malformation at this time.

New trials open frequently. Follow this disease to get notified.

Search ClinicalTrials.gov ↗Join the Genetic cardiac malformation community →

Specialists

22 foundView all specialists →
HM
Heymut Omran, MD
Specialist
PI on 1 active trial
DA
Dr. Evdokia Anagnostou
Specialist
PI on 1 active trial
KS
Kim G Nielsen, Dr Med Sci
Specialist
PI on 1 active trial
AM
Aniket Saha, MD
GREENVILLE, SC
Specialist
PI on 1 active trial1 Genetic cardiac malformation publication
WP
William T Cade, PT, PhD
HARKER HEIGHTS, TX
Specialist
PI on 5 active trials
LP
Lise Duranteau, MD, PhD
Specialist
PI on 1 active trial
RM
Rainard Fuhr, Dr. med.
Specialist
PI on 2 active trials
LP
Leandro Slipczuk, MD, PhD
Specialist
PI on 1 active trial
ID
Ipsen Medical, Director
Specialist
PI on 5 active trials
CP
Claus H Gravholt, Professor
Specialist
PI on 2 active trials
AM
Allan T Bombard, MD
SAN DIEGO, CA
Specialist
PI on 1 active trial1 Genetic cardiac malformation publication
CP
Chiara Di Resta, PhD
Specialist
PI on 1 active trial
TM
Thomas M. Egan, MD, MSc.
Specialist
PI on 2 active trials
GM
Galip Can Uyar, MD
Specialist
PI on 3 active trials1 Genetic cardiac malformation publication
AM
Aarti Sharma, MD
Specialist
PI on 1 active trial1 Genetic cardiac malformation publication
JN
James Neaton
Specialist
PI on 1 active trial1 Genetic cardiac malformation publication
RM
Romain Lazor, MD
Specialist
PI on 1 active trial
LT
Lloyd Taylor
Specialist
PI on 1 active trial27 Genetic cardiac malformation publications

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 Genetic cardiac malformation.

Search all travel grants →NORD Financial Assistance ↗

Community

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Latest news about Genetic cardiac malformation

No recent news articles for Genetic cardiac malformation.

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Caregiver Resources

NORD Caregiver Resources

Support, advocacy, and financial assistance for caregivers of rare disease patients.

Mental Health Support

Rare disease caregiving can be isolating. Connect with counseling and peer support.

Family & Caregiver Grants

Financial assistance programs specifically for caregivers of rare disease patients.

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.What specific type of heart defect does my child (or I) have, and what is causing it genetically?,Does my child need surgery or another procedure, and if so, when?,What physical activities are safe, and are there any I should avoid?,Should other family members be tested for the same genetic condition?,What signs should I watch for at home that mean I need to go to the emergency room?,Will my child need lifelong follow-up with a cardiologist, and what does that look like as they grow up?,Are there clinical trials or new treatments I should know about?

Common questions about Genetic cardiac malformation

What is Genetic cardiac malformation?

Genetic cardiac malformation (Orphanet code 477805) is a broad term used to describe heart defects that are caused — at least in part — by changes in a person's genes. These malformations happen when the heart does not form correctly during early development in the womb. The result can be structural problems such as holes between the heart's chambers, abnormal valves, or vessels that are connected in the wrong way. Some people also hear this group of conditions called 'congenital heart defects with a genetic cause' or 'hereditary congenital heart disease.' Because this is a category covering

Which specialists treat Genetic cardiac malformation?

22 specialists and care centers treating Genetic cardiac malformation are listed on UniteRare, sourced from ClinicalTrials.gov principal investigators, published research, and the NPPES NPI registry.