Leukoencephalopathy-palmoplantar keratoderma syndrome

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Overview

Leukoencephalopathy-palmoplantar keratoderma syndrome is an extremely rare genetic condition that affects both the brain's white matter and the skin. The name describes its two main features: leukoencephalopathy, which means damage or abnormal development of the white matter in the brain, and palmoplantar keratoderma, which refers to thickened, hardened skin on the palms of the hands and soles of the feet. White matter is the tissue in the brain that helps nerve cells communicate with each other, so when it is damaged, people may experience neurological problems such as difficulty with movement, coordination, thinking, and speech. The skin changes typically appear as thick, rough patches on the hands and feet that can be uncomfortable or painful. Neurological symptoms may include progressive difficulty walking, muscle stiffness or spasticity, and cognitive decline over time. The severity and progression of symptoms can vary between affected individuals. Because this condition is so rare, treatment options are currently limited to managing symptoms. There is no known cure. Care typically involves a team of specialists who address both the neurological and skin-related aspects of the disease. Physical therapy, medications for spasticity, and skin care treatments may help improve quality of life.

Key symptoms:

Thickened, hardened skin on palms and solesDifficulty walking or unsteady gaitMuscle stiffness or spasticityProgressive decline in thinking and memorySpeech difficultiesLoss of coordinationWhite matter abnormalities on brain MRIWeakness in the arms or legsDifficulty with fine motor tasksNumbness or tingling in hands and feet

Clinical phenotype terms (16)— hover any for plain English
Pseudobulbar signsHP:0002200TetraparesisHP:0002273EEG with generalized slow activityHP:0010845Diaphyseal dysplasiaHP:0100252Decreased number of peripheral myelinated nerve fibersHP:0003380
Inheritance

Autosomal recessive

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

Age of Onset

Variable

Can begin at different ages, from infancy through adulthood

Orphanet ↗NORD ↗

Treatments

No FDA-approved treatments are currently listed for Leukoencephalopathy-palmoplantar keratoderma syndrome.

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No actively recruiting trials found for Leukoencephalopathy-palmoplantar keratoderma syndrome at this time.

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No specialists are currently listed for Leukoencephalopathy-palmoplantar keratoderma 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 Leukoencephalopathy-palmoplantar keratoderma syndrome.

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Community

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Latest news about Leukoencephalopathy-palmoplantar keratoderma syndrome

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Caregiver Resources

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Mental Health Support

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Family & Caregiver Grants

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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.How severe is the white matter involvement on my brain MRI, and what does that mean for my future?,What treatments can help manage the thickened skin on my hands and feet?,Should I have genetic testing, and would it help my family members understand their risk?,How often should I have follow-up brain MRI scans and neurological evaluations?,What therapies (physical, occupational, speech) would benefit me most right now?,Are there any clinical trials or research studies I could participate in?,What should I watch for that would require urgent medical attention?

Common questions about Leukoencephalopathy-palmoplantar keratoderma syndrome

What is Leukoencephalopathy-palmoplantar keratoderma syndrome?

Leukoencephalopathy-palmoplantar keratoderma syndrome is an extremely rare genetic condition that affects both the brain's white matter and the skin. The name describes its two main features: leukoencephalopathy, which means damage or abnormal development of the white matter in the brain, and palmoplantar keratoderma, which refers to thickened, hardened skin on the palms of the hands and soles of the feet. White matter is the tissue in the brain that helps nerve cells communicate with each other, so when it is damaged, people may experience neurological problems such as difficulty with movemen

How is Leukoencephalopathy-palmoplantar keratoderma syndrome inherited?

Leukoencephalopathy-palmoplantar keratoderma syndrome follows a autosomal recessive inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.