Craniofacial dysostosis-diaphyseal hyperplasia syndrome

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ORPHA:1798OMIM:122900Q78.8
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Overview

Craniofacial dysostosis-diaphyseal hyperplasia syndrome is an extremely rare genetic bone disorder that affects both the skull and the long bones of the body. The name describes its two main features: 'craniofacial dysostosis' refers to abnormal development of the bones of the skull and face, while 'diaphyseal hyperplasia' means there is excessive bone growth along the shafts (middle portions) of the long bones in the arms and legs. This condition is sometimes also referred to as Craniofacial dysostosis with diaphyseal hyperplasia or by its Orphanet designation ORPHA:1798. Patients with this syndrome typically show abnormal skull shape, widely spaced eyes, a flat midface, and thickening of the long bones visible on X-rays. The overgrowth of bone in the skull can sometimes lead to compression of nerves, which may cause hearing loss or vision problems. Other features can include short stature, dental abnormalities, and limb differences. Because this condition is so rare, there is no specific cure or targeted therapy available. Treatment is supportive and focuses on managing individual symptoms. This may include surgery to relieve pressure on nerves caused by bone overgrowth, hearing aids if hearing is affected, dental care, and orthopedic management for bone-related complications. A team of specialists is usually needed to provide comprehensive care. Research into the genetic basis of this condition is ongoing, and understanding the exact gene involved may open doors to more targeted treatments in the future.

Also known as:

Key symptoms:

Abnormal skull shapeWidely spaced eyesFlat or underdeveloped midfaceThickened long bonesShort statureHearing lossVision problemsDental abnormalitiesProminent foreheadLimb abnormalitiesNerve compression from bone overgrowthBroad nasal bridgeJoint stiffness

Clinical phenotype terms (36)— hover any for plain English
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 Craniofacial dysostosis-diaphyseal hyperplasia syndrome.

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No actively recruiting trials found for Craniofacial dysostosis-diaphyseal hyperplasia syndrome at this time.

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No specialists are currently listed for Craniofacial dysostosis-diaphyseal hyperplasia 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 Craniofacial dysostosis-diaphyseal hyperplasia syndrome.

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Community

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Caregiver Resources

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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.What specific bones are affected in my child's case, and how severe is the involvement?,How often should we schedule imaging studies to monitor bone growth?,Are there any signs of nerve compression that we should watch for at home?,Should we pursue genetic testing, and what type would be most informative?,What surgical options might be needed now or in the future?,How can we best support hearing and vision if they become affected?,Are there any clinical trials or research studies we could participate in?

Common questions about Craniofacial dysostosis-diaphyseal hyperplasia syndrome

What is Craniofacial dysostosis-diaphyseal hyperplasia syndrome?

Craniofacial dysostosis-diaphyseal hyperplasia syndrome is an extremely rare genetic bone disorder that affects both the skull and the long bones of the body. The name describes its two main features: 'craniofacial dysostosis' refers to abnormal development of the bones of the skull and face, while 'diaphyseal hyperplasia' means there is excessive bone growth along the shafts (middle portions) of the long bones in the arms and legs. This condition is sometimes also referred to as Craniofacial dysostosis with diaphyseal hyperplasia or by its Orphanet designation ORPHA:1798. Patients with this

How is Craniofacial dysostosis-diaphyseal hyperplasia syndrome inherited?

Craniofacial dysostosis-diaphyseal hyperplasia syndrome follows a autosomal dominant inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.

At what age does Craniofacial dysostosis-diaphyseal hyperplasia syndrome typically begin?

Typical onset of Craniofacial dysostosis-diaphyseal hyperplasia syndrome is neonatal. Age of onset can vary across affected individuals.