Dysostosis with brachydactyly without extraskeletal manifestations

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ORPHA:498451
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Overview

Dysostosis with brachydactyly without extraskeletal manifestations is a rare inherited bone disorder that mainly affects the hands and feet. 'Dysostosis' means that certain bones did not form properly during development, and 'brachydactyly' means short fingers or toes. The phrase 'without extraskeletal manifestations' means that the condition is limited to the skeleton — it does not affect the heart, brain, eyes, or other internal organs. This makes it distinct from many other similar conditions that can involve multiple body systems. People with this condition are typically born with noticeably short fingers and/or toes, and some bones in the hands or feet may be unusually shaped or fused together. The condition is usually identified at birth or in early childhood when the hand and foot differences become visible. In most cases, intelligence and overall health are not affected. Treatment is focused on managing symptoms and improving hand and foot function. Depending on how severe the bone changes are, some people may benefit from physical or occupational therapy, and in certain cases surgery may be considered to improve movement or appearance. There is no cure, but many people with this condition live full, healthy lives with normal life expectancy.

Key symptoms:

Short fingers (brachydactyly)Short toesUnusually shaped bones in the hands or feetFused or missing finger or toe bonesReduced grip strength or hand functionDifficulty with fine motor tasks like writing or buttoningShortened metacarpal or metatarsal bones (the bones in the palm or sole)Asymmetry between hands or feet in some cases

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 ↗NORD ↗

Treatments

No FDA-approved treatments are currently listed for Dysostosis with brachydactyly without extraskeletal manifestations.

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No actively recruiting trials found for Dysostosis with brachydactyly without extraskeletal manifestations at this time.

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No specialists are currently listed for Dysostosis with brachydactyly without extraskeletal manifestations.

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 Dysostosis with brachydactyly without extraskeletal manifestations.

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Community

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Caregiver Resources

NORD Caregiver Resources

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Social Security Disability

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Questions for your doctor

Bring these to your next appointment

  • Q1.Which specific bones are affected in my child's hands and feet, and how severe is it?,Should we do genetic testing, and which genes should be tested?,Will my child's hand function improve over time, or stay the same?,Is surgery recommended, and if so, what is the best timing?,What therapies would help most with daily tasks and school activities?,Are other family members at risk, and should they be tested?,Are there any specialists or centers with specific experience in this condition that you would recommend?

Common questions about Dysostosis with brachydactyly without extraskeletal manifestations

What is Dysostosis with brachydactyly without extraskeletal manifestations?

Dysostosis with brachydactyly without extraskeletal manifestations is a rare inherited bone disorder that mainly affects the hands and feet. 'Dysostosis' means that certain bones did not form properly during development, and 'brachydactyly' means short fingers or toes. The phrase 'without extraskeletal manifestations' means that the condition is limited to the skeleton — it does not affect the heart, brain, eyes, or other internal organs. This makes it distinct from many other similar conditions that can involve multiple body systems. People with this condition are typically born with noticea

How is Dysostosis with brachydactyly without extraskeletal manifestations inherited?

Dysostosis with brachydactyly without extraskeletal manifestations follows a autosomal dominant inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.

At what age does Dysostosis with brachydactyly without extraskeletal manifestations typically begin?

Typical onset of Dysostosis with brachydactyly without extraskeletal manifestations is neonatal. Age of onset can vary across affected individuals.