Keppen-Lubinsky syndrome

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ORPHA:435628OMIM:614098E88.1
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Overview

Keppen-Lubinsky syndrome (also known as KLS) is an extremely rare genetic condition that affects multiple body systems. It was first described in 1997 and is caused by changes (mutations) in the KCNJ6 gene. The syndrome is characterized by a distinctive combination of features present from birth, including a very lean body with almost no body fat (called lipodystrophy), tight skin that appears aged or wrinkled, and distinctive facial features. Affected individuals typically have a narrow face, prominent eyes, a thin nose, and a small mouth with a high-arched palate. Intellectual disability and developmental delays are common features of this condition. Babies born with Keppen-Lubinsky syndrome often have significant feeding difficulties, poor growth, and low muscle tone (hypotonia). Many affected children also experience hearing loss and may have joint contractures, where the joints become stiff and difficult to move. The condition can also affect the nervous system, leading to problems with movement and coordination. There is currently no cure for Keppen-Lubinsky syndrome. Treatment focuses on managing individual symptoms and supporting the child's development. This may include nutritional support, physical therapy, occupational therapy, speech therapy, and hearing aids if needed. Because the condition is so rare, with only a handful of cases reported worldwide, knowledge about the full range of symptoms and long-term outcomes is still growing.

Also known as:

Key symptoms:

Very little body fat (lipodystrophy)Tight, wrinkled skin that looks agedDistinctive narrow face with prominent eyesIntellectual disabilityDevelopmental delaysFeeding difficulties in infancyPoor growth and failure to thriveLow muscle tone (floppiness)Hearing lossStiff joints or joint contracturesHigh-arched palateSmall mouthThin noseDelayed speech developmentMovement and coordination problems

Clinical phenotype terms (42)— hover any for plain English
Progeroid facial appearanceHP:0005328Congenital generalized lipodystrophyHP:0009059Premature skin wrinklingHP:0100678Abnormal forehead morphologyHP:0000290Loss of facial adipose tissueHP:0000292Shallow orbitsHP:0000586Abnormally large globeHP:0001090
Inheritance

Autosomal dominant

Passed on from just one parent; each child has about a 50% chance of inheriting it

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 Keppen-Lubinsky syndrome.

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No actively recruiting trials found for Keppen-Lubinsky syndrome at this time.

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

Search ClinicalTrials.gov ↗Join the Keppen-Lubinsky syndrome community →

No specialists are currently listed for Keppen-Lubinsky 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 Keppen-Lubinsky syndrome.

Search all travel grants →NORD Financial Assistance ↗

Community

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Latest news about Keppen-Lubinsky syndrome

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Caregiver Resources

NORD Caregiver Resources

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

Mental Health Support

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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 KCNJ6 mutation does my child have, and what does it mean for their health?,What therapies and interventions should we start right away?,How should we manage feeding difficulties, and will my child need tube feeding?,What developmental milestones should we watch for, and when should we be concerned?,Should my child have a hearing test, and how often should hearing be monitored?,Are there any clinical trials or research studies we could participate in?,What is the chance of this condition occurring again in future pregnancies?

Common questions about Keppen-Lubinsky syndrome

What is Keppen-Lubinsky syndrome?

Keppen-Lubinsky syndrome (also known as KLS) is an extremely rare genetic condition that affects multiple body systems. It was first described in 1997 and is caused by changes (mutations) in the KCNJ6 gene. The syndrome is characterized by a distinctive combination of features present from birth, including a very lean body with almost no body fat (called lipodystrophy), tight skin that appears aged or wrinkled, and distinctive facial features. Affected individuals typically have a narrow face, prominent eyes, a thin nose, and a small mouth with a high-arched palate. Intellectual disability and

How is Keppen-Lubinsky syndrome inherited?

Keppen-Lubinsky syndrome follows a autosomal dominant inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.

At what age does Keppen-Lubinsky syndrome typically begin?

Typical onset of Keppen-Lubinsky syndrome is neonatal. Age of onset can vary across affected individuals.