Kallmann syndrome-heart disease syndrome

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Overview

Kallmann syndrome-heart disease syndrome is an extremely rare condition that combines features of Kallmann syndrome with congenital heart defects. Kallmann syndrome itself is a condition where the body does not produce enough gonadotropin-releasing hormone (GnRH), which leads to delayed or absent puberty and a reduced or absent sense of smell (called anosmia or hyposmia). In this particular syndrome, these hormonal and smell-related problems occur alongside structural heart abnormalities that are present from birth. People with this condition may notice that puberty does not start on its own, and they may have difficulty detecting odors. The heart defects can vary in type and severity, and may include problems with the heart's structure such as septal defects or other congenital malformations. Some individuals may also have additional features such as hearing loss or other developmental differences. Treatment typically involves hormone replacement therapy to bring on puberty and support normal sexual development and bone health. Heart defects may require monitoring by a cardiologist, and in some cases, surgical correction may be needed. Because this syndrome is so rare, management is usually coordinated by a team of specialists who tailor treatment to each person's specific combination of symptoms. Early diagnosis and intervention can significantly improve quality of life and long-term outcomes.

Key symptoms:

Delayed or absent pubertyReduced or absent sense of smellCongenital heart defectsUnderdeveloped genitaliaInfertility or reduced fertilityAbsent or irregular menstrual periods in femalesSmall testes in malesPossible hearing lossLow sex hormone levelsPossible cleft lip or palatePossible kidney abnormalities

Clinical phenotype terms (29)— hover any for plain English
Total anosmiaHP:0010632Short lingual frenulumHP:0000200Double outlet right ventricleHP:0001719Pulmonary artery hypoplasiaHP:0004971Bilateral cryptorchidismHP:0008689
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 Kallmann syndrome-heart disease syndrome.

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No actively recruiting trials found for Kallmann syndrome-heart disease syndrome at this time.

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Search ClinicalTrials.gov ↗Join the Kallmann syndrome-heart disease syndrome community →

No specialists are currently listed for Kallmann syndrome-heart disease syndrome.

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 Kallmann syndrome-heart disease syndrome.

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Community

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Latest news about Kallmann syndrome-heart disease syndrome

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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 type of heart defect does my child or I have, and how severe is it?,When should hormone replacement therapy be started, and what are the options?,Will fertility be possible in the future, and what treatments might help?,How often do we need cardiac check-ups and hormone level monitoring?,Are there any physical activities or sports that should be avoided?,Should other family members be tested for this condition?,What genetic testing is recommended, and what can the results tell us?

Common questions about Kallmann syndrome-heart disease syndrome

What is Kallmann syndrome-heart disease syndrome?

Kallmann syndrome-heart disease syndrome is an extremely rare condition that combines features of Kallmann syndrome with congenital heart defects. Kallmann syndrome itself is a condition where the body does not produce enough gonadotropin-releasing hormone (GnRH), which leads to delayed or absent puberty and a reduced or absent sense of smell (called anosmia or hyposmia). In this particular syndrome, these hormonal and smell-related problems occur alongside structural heart abnormalities that are present from birth. People with this condition may notice that puberty does not start on its own,