Mazabraud syndrome

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ORPHA:57782M85.0
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Overview

Mazabraud syndrome is a very rare condition where two things happen together in the same person: fibrous dysplasia of bone and soft tissue myxomas. Fibrous dysplasia means that normal bone tissue is replaced by a weaker, fibrous (scar-like) material, which can cause bones to become misshapen, painful, or prone to fracturing. Myxomas are benign (non-cancerous) jelly-like tumors that grow in the muscles near the affected bones. The syndrome was first described by French physician André Mazabraud in the 1960s, and it is sometimes called Mazabraud's syndrome. The bones most commonly affected include the thigh bone (femur), shin bone (tibia), and the bones of the skull and face, though any bone can be involved. The myxomas usually appear in the muscles closest to the diseased bone. People with this condition may experience bone pain, limping, repeated fractures, and swelling or lumps in the muscles. The severity can range widely from person to person. Treatment focuses on managing symptoms rather than curing the disease. Surgery is often used to stabilize weakened bones or remove troublesome myxomas. There is no approved drug that reverses the underlying bone changes, though bisphosphonate medications are sometimes used to help reduce bone pain and slow bone loss. Regular monitoring by a team of specialists is an important part of care.

Also known as:

Key symptoms:

Bone pain, especially in the legs, hips, or skullRepeated or unusual bone fracturesBones that become bowed or misshapen over timeSoft, painless lumps or swellings in the musclesLimping or difficulty walkingLeg length difference if the thigh or shin bone is affectedFacial asymmetry if skull or facial bones are involvedMuscle weakness near affected areasSwelling around the affected bone or muscle

Clinical phenotype terms (5)— hover any for plain English
Fibrous dysplasia of the bonesHP:0010734
Inheritance

Sporadic

Usually appears on its own, not inherited from a parent

Age of Onset

Variable

Can begin at different ages, from infancy through adulthood

Orphanet ↗NORD ↗

Treatments

No FDA-approved treatments are currently listed for Mazabraud syndrome.

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No actively recruiting trials found for Mazabraud syndrome at this time.

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Search ClinicalTrials.gov ↗Join the Mazabraud syndrome community →

No specialists are currently listed for Mazabraud 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 Mazabraud syndrome.

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Community

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Latest news about Mazabraud 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.How many bones are affected in my case, and which ones are at highest risk for fracture?,Should I have genetic testing of my tumor tissue to confirm the GNAS mutation?,What imaging schedule do you recommend to monitor my bones and myxomas over time?,Are bisphosphonate medications appropriate for me, and what are the risks and benefits?,When should a myxoma be removed, and what is the chance it will come back after surgery?,What warning signs should prompt me to seek emergency care?,Are there any clinical trials or research studies I could participate in?

Common questions about Mazabraud syndrome

What is Mazabraud syndrome?

Mazabraud syndrome is a very rare condition where two things happen together in the same person: fibrous dysplasia of bone and soft tissue myxomas. Fibrous dysplasia means that normal bone tissue is replaced by a weaker, fibrous (scar-like) material, which can cause bones to become misshapen, painful, or prone to fracturing. Myxomas are benign (non-cancerous) jelly-like tumors that grow in the muscles near the affected bones. The syndrome was first described by French physician André Mazabraud in the 1960s, and it is sometimes called Mazabraud's syndrome. The bones most commonly affected incl

How is Mazabraud syndrome inherited?

Mazabraud syndrome follows a sporadic inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.