Dysostosis with brachydactyly with extraskeletal manifestations

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ORPHA:498454
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Overview

Dysostosis with brachydactyly with extraskeletal manifestations is a very rare genetic condition that affects the bones, particularly the hands and feet, along with other parts of the body. 'Dysostosis' means that certain bones did not form properly during development, and 'brachydactyly' means that the fingers and toes are unusually short. Together, these features cause noticeable differences in the shape and length of the hands and feet from birth or early childhood. Beyond the skeleton, this condition can also affect other body systems, which is why it is described as having 'extraskeletal manifestations.' These may include differences in facial features, growth, and possibly other organs depending on the specific genetic cause in each person. Because this is an extremely rare condition, much of what is known comes from a small number of reported cases. There is currently no cure, and treatment focuses on managing individual symptoms and improving quality of life. A team of specialists is usually needed to address the different ways this condition can affect the body. Early diagnosis through genetic testing is important so that the right support and monitoring can be put in place as soon as possible.

Key symptoms:

Short fingers and toes (brachydactyly)Abnormally shaped or fused bones in the hands and feetDistinctive facial features such as a flat face or wide-set eyesShort stature or slower than usual growthDifferences in the shape of the skull or facePossible intellectual disability or learning difficultiesJoint stiffness or limited movement in the fingersAbnormalities in other bones of the bodyPossible heart or organ differences (extraskeletal features)

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 with extraskeletal manifestations.

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No actively recruiting trials found for Dysostosis with brachydactyly with extraskeletal manifestations at this time.

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Search ClinicalTrials.gov ↗Join the Dysostosis with brachydactyly with extraskeletal manifestations community →

No specialists are currently listed for Dysostosis with brachydactyly with 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 with extraskeletal manifestations.

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Community

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Latest news about Dysostosis with brachydactyly with extraskeletal manifestations

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Caregiver Resources

NORD Caregiver Resources

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Mental Health Support

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Family & Caregiver Grants

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Social Security Disability

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Questions for your doctor

Bring these to your next appointment

  • Q1.What specific gene or genetic change is causing this condition in my child, and has it been identified yet?,Which body systems should we be monitoring beyond the bones and hands?,Would surgery on the hands or feet be helpful, and if so, when is the best time to consider it?,What therapies are available to help my child with daily tasks and school activities?,Are there other families or specialists who have experience with this specific condition?,What should I watch for that would mean my child needs to be seen urgently?,Are other family members at risk, and should they be tested?

Common questions about Dysostosis with brachydactyly with extraskeletal manifestations

What is Dysostosis with brachydactyly with extraskeletal manifestations?

Dysostosis with brachydactyly with extraskeletal manifestations is a very rare genetic condition that affects the bones, particularly the hands and feet, along with other parts of the body. 'Dysostosis' means that certain bones did not form properly during development, and 'brachydactyly' means that the fingers and toes are unusually short. Together, these features cause noticeable differences in the shape and length of the hands and feet from birth or early childhood. Beyond the skeleton, this condition can also affect other body systems, which is why it is described as having 'extraskeletal

How is Dysostosis with brachydactyly with extraskeletal manifestations inherited?

Dysostosis with brachydactyly with 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 with extraskeletal manifestations typically begin?

Typical onset of Dysostosis with brachydactyly with extraskeletal manifestations is neonatal. Age of onset can vary across affected individuals.