Congenital vascular bone syndrome

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ORPHA:235832
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17Specialists8Treatment centers

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UniteRare data is sourced from FDA.gov, ClinicalTrials.gov, Orphanet, OMIM, and NORD.
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Overview

Congenital vascular bone syndrome is an extremely rare condition that is present from birth and involves abnormal blood vessel formation (vascular malformations) that affects the bones. In this syndrome, unusual blood vessels develop in or around bones, which can lead to problems with bone growth, bone pain, and changes in the shape or strength of the affected bones. The vascular malformations may cause swelling, warmth, or discoloration of the skin over the affected area. Depending on which bones are involved, patients may experience differences in limb length, fractures, or difficulty with movement. Because this condition is so rare, the medical understanding of it is still limited. Diagnosis typically involves a combination of imaging studies such as X-rays, MRI scans, and sometimes CT scans to see both the bone changes and the abnormal blood vessels. Treatment is generally focused on managing symptoms rather than curing the condition. This may include pain management, orthopedic interventions to address bone deformities or fractures, and sometimes procedures to treat the vascular malformations themselves, such as embolization (blocking abnormal blood vessels) or surgery. A team of specialists is usually needed to provide the best care. Because so few cases have been described, there is no single established treatment protocol, and care is often tailored to each individual patient's needs.

Key symptoms:

Abnormal blood vessel growths in or near bonesBone painBone deformitiesSwelling over affected bonesDifferences in limb lengthSkin discoloration or birthmarks over affected areasWarmth over affected areasIncreased risk of bone fracturesDifficulty with movement or walkingAbnormal bone growth

Inheritance

Variable

Can be inherited in different ways depending on the underlying gene

Age of Onset

Neonatal

Begins at or shortly after birth (first 4 weeks)

Orphanet ↗NORD ↗

Treatments

No FDA-approved treatments are currently listed for Congenital vascular bone syndrome.

View clinical trials →

No actively recruiting trials found for Congenital vascular bone syndrome at this time.

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

Search ClinicalTrials.gov ↗Join the Congenital vascular bone syndrome community →

Specialists

17 foundView all specialists →
TM
Thomas G. Martin, MD
Specialist
PI on 4 active trials
CD
Colleen Delaney
OLD GREENWICH, CT
Specialist
PI on 3 active trials
PM
Paul Martin
Specialist
PI on 3 active trials73 Congenital vascular bone syndrome publications
MM
Melisa Ruiz-Gutierrez, M.D.
BOSTON, MA
Specialist
PI on 1 active trial
MM
Meral Gunay-Aygun, M.D.
BETHESDA, MD
Specialist
PI on 2 active trials
AW
Ann Woolfrey
SEATTLE, WA
Specialist
PI on 5 active trials
YM
Yi-Bin A Chen, M.D.
BOSTON, MA
Specialist
PI on 1 active trial
PM
Paul Armistead, MD
CHAPEL HILL, NC
Specialist
PI on 1 active trial
AM
Aarti Sharma, MD
Specialist
PI on 1 active trial1 Congenital vascular bone syndrome publication
IV
Indumathy Varadarajan
CHARLOTTESVILLE, VA
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 Congenital vascular bone syndrome.

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Community

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Latest news about Congenital vascular bone 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.Which bones and blood vessels are affected in my child's case?,What imaging or tests will be needed to monitor the condition over time?,Are there any activities my child should avoid to reduce the risk of fractures?,What treatment options are available for the vascular malformations?,Should we consider genetic testing to look for an underlying cause?,What specialists should be part of our care team?,Are there any clinical trials or research studies we could participate in?

Common questions about Congenital vascular bone syndrome

What is Congenital vascular bone syndrome?

Congenital vascular bone syndrome is an extremely rare condition that is present from birth and involves abnormal blood vessel formation (vascular malformations) that affects the bones. In this syndrome, unusual blood vessels develop in or around bones, which can lead to problems with bone growth, bone pain, and changes in the shape or strength of the affected bones. The vascular malformations may cause swelling, warmth, or discoloration of the skin over the affected area. Depending on which bones are involved, patients may experience differences in limb length, fractures, or difficulty with m

At what age does Congenital vascular bone syndrome typically begin?

Typical onset of Congenital vascular bone syndrome is neonatal. Age of onset can vary across affected individuals.

Which specialists treat Congenital vascular bone syndrome?

17 specialists and care centers treating Congenital vascular bone syndrome are listed on UniteRare, sourced from ClinicalTrials.gov principal investigators, published research, and the NPPES NPI registry.