Kufor-Rakeb syndrome

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ORPHA:306674OMIM:606693G23.0
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2Specialists8Treatment centers

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Overview

Kufor-Rakeb syndrome, also known as PARK9 or Pallido-pyramidal syndrome, is a very rare inherited neurological disorder that affects the brain and nervous system. It is named after the village in Jordan where it was first described. The condition belongs to a group of disorders called neurodegeneration with brain iron accumulation (NBIA), because iron tends to build up in certain parts of the brain over time. Kufor-Rakeb syndrome typically begins in the teenage years and causes symptoms similar to Parkinson's disease, but at a much younger age. People with this condition develop stiffness and slowness of movement (parkinsonism), difficulty walking, muscle spasms, and problems with eye movements. Over time, many individuals also experience intellectual decline (dementia), difficulty speaking and swallowing, and involuntary facial movements. Some people develop visual hallucinations. There is currently no cure for Kufor-Rakeb syndrome. Treatment focuses on managing symptoms. Levodopa, a medication commonly used for Parkinson's disease, may help with movement problems in the early stages, but its effectiveness often decreases over time. Physical therapy, speech therapy, and occupational therapy are important parts of ongoing care. Research is continuing to better understand this condition and find more effective treatments.

Also known as:

Key symptoms:

Stiffness and slowness of movement (parkinsonism)Difficulty walking and balance problemsMuscle spasms and rigidityAbnormal eye movements, especially difficulty looking upwardIntellectual decline and dementiaDifficulty speaking clearlyDifficulty swallowingInvoluntary facial or jaw movementsVisual hallucinationsTremorLoss of facial expression (mask-like face)Behavioral and psychiatric changesMini-strokes or mini-seizure-like episodesProgressive loss of independence

Clinical phenotype terms (44)— hover any for plain English
Slow saccadic eye movementsHP:0000514Supranuclear gaze palsyHP:0000605Tongue muscle weaknessHP:0000183Visual hallucinationHP:0002367
Inheritance

Autosomal recessive

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

Age of Onset

Juvenile

Begins in the teen years

Orphanet ↗OMIM ↗NORD ↗

Treatments

No FDA-approved treatments are currently listed for Kufor-Rakeb syndrome.

View clinical trials →

No actively recruiting trials found for Kufor-Rakeb syndrome at this time.

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

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Specialists

2 foundView all specialists →
TD
Thomas Klopstock, Prof. Dr.
Specialist
PI on 3 active trials
AP
Angela Schulz, MD, PhD
Hamburg
Specialist

Rare Disease Specialist

PI on 1 active trial

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 Kufor-Rakeb syndrome.

Search all travel grants →NORD Financial Assistance ↗

Community

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Latest news about Kufor-Rakeb 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.How quickly do you expect symptoms to progress in my case?,Is levodopa likely to help, and for how long might it remain effective?,What therapies (physical, speech, occupational) should we start now?,Are there any clinical trials or research studies I could participate in?,What signs should I watch for that would need urgent medical attention?,How should we plan for changes in cognitive function over time?,Can you refer us to a genetic counselor to discuss family planning and testing for other family members?

Common questions about Kufor-Rakeb syndrome

What is Kufor-Rakeb syndrome?

Kufor-Rakeb syndrome, also known as PARK9 or Pallido-pyramidal syndrome, is a very rare inherited neurological disorder that affects the brain and nervous system. It is named after the village in Jordan where it was first described. The condition belongs to a group of disorders called neurodegeneration with brain iron accumulation (NBIA), because iron tends to build up in certain parts of the brain over time. Kufor-Rakeb syndrome typically begins in the teenage years and causes symptoms similar to Parkinson's disease, but at a much younger age. People with this condition develop stiffness and

How is Kufor-Rakeb syndrome inherited?

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

At what age does Kufor-Rakeb syndrome typically begin?

Typical onset of Kufor-Rakeb syndrome is juvenile. Age of onset can vary across affected individuals.

Which specialists treat Kufor-Rakeb syndrome?

2 specialists and care centers treating Kufor-Rakeb syndrome are listed on UniteRare, sourced from ClinicalTrials.gov principal investigators, published research, and the NPPES NPI registry.