Osteosclerotic bone dysplasia

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ORPHA:1832OMIM:259775Q78.2
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4Specialists8Treatment centers

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

Osteosclerotic bone dysplasia (also sometimes referred to as osteosclerotic skeletal dysplasia) is a very rare genetic condition that affects how bones develop and remodel. In this disorder, the bones become abnormally dense and hard — a process called osteosclerosis. Normally, bone is constantly being broken down and rebuilt in a balanced cycle. In osteosclerotic bone dysplasia, this balance is disrupted, leading to excessive bone density. While denser bones might sound like a good thing, the overly hardened bone is often brittle and structurally abnormal, which can lead to a range of problems. Key features of this condition can include skeletal abnormalities visible on X-rays, bone pain, increased risk of fractures despite the dense appearance of bones, and in some cases narrowing of the spaces within bones where nerves and blood vessels pass. This narrowing can lead to nerve compression, causing symptoms like hearing loss, vision problems, or facial nerve weakness. Some individuals may also experience problems with bone marrow function because the marrow space inside bones becomes crowded out by the excess bone tissue, potentially leading to low blood counts (anemia or other cytopenias). There is currently no cure for osteosclerotic bone dysplasia. Treatment is mainly supportive and focused on managing symptoms. This may include pain management, surgical intervention for fractures or nerve compression, and monitoring of blood counts. Because this condition is extremely rare, management is best guided by a team of specialists familiar with skeletal dysplasias.

Also known as:

Key symptoms:

Abnormally dense bones seen on X-raysBone painIncreased risk of bone fracturesHearing loss due to nerve compressionVision problems from narrowed nerve channelsFacial nerve weakness or paralysisLow blood counts (anemia)Short stature or growth problemsSkeletal deformitiesFrequent infections if immune cells are affectedDental problemsHeadachesEnlarged liver or spleen

Clinical phenotype terms (20)— hover any for plain English
Gingival fibromatosisHP:0000169Mandibular aplasiaHP:0009939Median cleft palateHP:0009099
Inheritance

Variable

Can be inherited in different ways depending on the underlying gene

Age of Onset

Variable

Can begin at different ages, from infancy through adulthood

Orphanet ↗OMIM ↗NORD ↗

FDA & Trial Timeline

2 events
Feb 2025RNS for Treatment-resistant Obsessive-compulsive Disorder

Xuanwu Hospital, Beijing — NA

TrialRECRUITING
Feb 2025Effect of RNS in Treatment-refractory Tourette's Syndrome

Xuanwu Hospital, Beijing — NA

TrialRECRUITING

Data sourced from FDA regulatory filings and ClinicalTrials.gov. Updated periodically.

Treatments

No FDA-approved treatments are currently listed for Osteosclerotic bone dysplasia.

View clinical trials →

No actively recruiting trials found for Osteosclerotic bone dysplasia at this time.

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

Search ClinicalTrials.gov ↗Join the Osteosclerotic bone dysplasia community →

Specialists

4 foundView all specialists →
TK
Toshiyasu Koike
Specialist
1 Osteosclerotic bone dysplasia publication
TM
Tadahisa Mikami
Specialist
1 Osteosclerotic bone dysplasia publication
JT
Jun-Ichi Tamura
Specialist
1 Osteosclerotic bone dysplasia publication
HK
Hiroshi Kitagawa
Specialist
1 Osteosclerotic bone dysplasia publication

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 Osteosclerotic bone dysplasia.

Search all travel grants →NORD Financial Assistance ↗

Community

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Latest news about Osteosclerotic bone dysplasia

Disease timeline:

New recruiting trial: Effect of RNS in Treatment-refractory Tourette's Syndrome

A new clinical trial is recruiting patients for Osteosclerotic bone dysplasia

New recruiting trial: RNS for Treatment-resistant Obsessive-compulsive Disorder

A new clinical trial is recruiting patients for Osteosclerotic bone dysplasia

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 type of osteosclerotic bone dysplasia does my child or I have, and is genetic testing available to confirm it?,How often should we have X-rays, blood tests, and hearing or vision screenings?,What are the warning signs of complications I should watch for at home?,Are there any activity restrictions to reduce the risk of fractures?,Is bone marrow transplant or any other advanced treatment an option in our case?,Are there any clinical trials or research studies we could participate in?,Can you refer us to a center with experience in skeletal dysplasias?

Common questions about Osteosclerotic bone dysplasia

What is Osteosclerotic bone dysplasia?

Osteosclerotic bone dysplasia (also sometimes referred to as osteosclerotic skeletal dysplasia) is a very rare genetic condition that affects how bones develop and remodel. In this disorder, the bones become abnormally dense and hard — a process called osteosclerosis. Normally, bone is constantly being broken down and rebuilt in a balanced cycle. In osteosclerotic bone dysplasia, this balance is disrupted, leading to excessive bone density. While denser bones might sound like a good thing, the overly hardened bone is often brittle and structurally abnormal, which can lead to a range of problem

Which specialists treat Osteosclerotic bone dysplasia?

4 specialists and care centers treating Osteosclerotic bone dysplasia are listed on UniteRare, sourced from ClinicalTrials.gov principal investigators, published research, and the NPPES NPI registry.