Eiken syndrome

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ORPHA:79106OMIM:600002M85.8
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Overview

Eiken syndrome is an extremely rare inherited bone disorder that affects how the skeleton grows and develops. It is also sometimes referred to as 'Eiken skeletal dysplasia.' The condition is caused by changes (mutations) in the PTH1R gene, which gives instructions for making a protein that helps regulate bone and cartilage growth. When this gene does not work properly, the bones develop abnormally, leading to a condition called delayed bone maturation — meaning the bones look much younger on X-ray than the person's actual age. The main features of Eiken syndrome include very delayed hardening (ossification) of the bones, unusual bone shapes, and changes in the growth plates — the areas at the ends of bones where growth happens in children. People with this condition may have short stature, joint problems, and abnormal bone structure throughout the body, including the spine, pelvis, and limbs. The hands and feet may also look different. Because Eiken syndrome is so rare, treatment is focused on managing symptoms rather than curing the underlying cause. Care typically involves a team of specialists including bone doctors, geneticists, and physical therapists. There is currently no approved drug that targets the root cause of the disease. Management aims to support mobility, reduce pain, and monitor bone health over time.

Key symptoms:

Very delayed bone hardening (ossification) compared to ageShort stature or slower than normal growthAbnormal bone shapes seen on X-rayChanges in the growth plates of bonesUnusual appearance of the pelvis and hip bonesAbnormal spine shape or structureDifferences in the shape of hands and feetJoint stiffness or limited range of motionMild to moderate skeletal deformitiesBones that appear much younger than the person's actual age on imaging

Clinical phenotype terms (23)— hover any for plain English
Epiphyseal dysplasiaHP:0002656Limited elbow flexionHP:0006376Broad palmHP:0001169Abnormal fingertip morphologyHP:0001211Broad footHP:0001769Delayed epiphyseal ossificationHP:0002663Thin bony cortexHP:0002753Metaphyseal irregularityHP:0003025Fibular hypoplasiaHP:0003038Abnormal acetabulum morphologyHP:0003170Narrow pelvis boneHP:0003275Limited hip movementHP:0008800High iliac wingHP:0008808Short phalanx of fingerHP:0009803
Inheritance

Autosomal recessive

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

Age of Onset

Childhood

Begins in childhood, roughly ages 1 to 12

Orphanet ↗OMIM ↗NORD ↗

Treatments

No FDA-approved treatments are currently listed for Eiken syndrome.

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No actively recruiting trials found for Eiken syndrome at this time.

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

Search ClinicalTrials.gov ↗Join the Eiken syndrome community →

No specialists are currently listed for Eiken 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 Eiken syndrome.

Search all travel grants →NORD Financial Assistance ↗

Community

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Latest news about Eiken 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 specific mutations were found in the PTH1R gene, and what do they mean for my child's health?,How often should we have X-rays or other imaging to monitor bone development?,What physical activities are safe, and are there any we should avoid?,Should other family members be tested for PTH1R mutations?,Are there any clinical trials or research studies we could participate in?,What signs should prompt us to seek urgent medical attention?,Can you refer us to a center that specializes in rare skeletal dysplasias?

Common questions about Eiken syndrome

What is Eiken syndrome?

Eiken syndrome is an extremely rare inherited bone disorder that affects how the skeleton grows and develops. It is also sometimes referred to as 'Eiken skeletal dysplasia.' The condition is caused by changes (mutations) in the PTH1R gene, which gives instructions for making a protein that helps regulate bone and cartilage growth. When this gene does not work properly, the bones develop abnormally, leading to a condition called delayed bone maturation — meaning the bones look much younger on X-ray than the person's actual age. The main features of Eiken syndrome include very delayed hardening

How is Eiken syndrome inherited?

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

At what age does Eiken syndrome typically begin?

Typical onset of Eiken syndrome is childhood. Age of onset can vary across affected individuals.