Familial normophosphatemic tumoral calcinosis

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ORPHA:306658OMIM:610455M11.2
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Overview

Familial normophosphatemic tumoral calcinosis is a rare inherited condition in which large deposits of calcium form in the soft tissues of the body, especially around joints. Unlike other forms of tumoral calcinosis, the phosphate levels in the blood remain normal (hence the term 'normophosphatemic'). The calcium deposits, sometimes called calcinotic masses, tend to appear around the hips, shoulders, elbows, knees, and other joints. These masses can grow quite large and may cause pain, limit movement, and sometimes break through the skin, leading to drainage of a chalky white material. The condition typically begins in childhood or adolescence and tends to worsen over time as more calcium deposits develop. The disease is caused by genetic changes that affect how the body handles calcium in tissues. Because phosphate levels are normal, this condition is distinct from the more common hyperphosphatemic tumoral calcinosis, which involves elevated blood phosphate. Treatment is mainly focused on managing symptoms. Surgical removal of the calcium masses is the most common approach, but deposits frequently come back after surgery. There is no widely accepted medication that reliably prevents new deposits from forming. Some patients may benefit from medications that reduce inflammation or calcium deposition, but results vary. Ongoing research aims to better understand the underlying mechanisms and develop more effective therapies.

Key symptoms:

Large firm lumps around jointsPainful swelling near hips, shoulders, elbows, or kneesLimited range of motion in affected jointsChalky white discharge draining from skin over calcium depositsSkin breakdown or ulceration over the lumpsRecurrent infections at sites where deposits break through the skinDifficulty walking or moving affected limbsStiffness around jointsCosmetic disfigurement from large massesNormal blood phosphate levels despite calcium deposits

Inheritance

Autosomal recessive

Passed on when both parents carry the same gene change; often skips generations

Age of Onset

Childhood

Begins in childhood, roughly ages 1 to 12

Orphanet ↗OMIM ↗NORD ↗

Treatments

No FDA-approved treatments are currently listed for Familial normophosphatemic tumoral calcinosis.

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No actively recruiting trials found for Familial normophosphatemic tumoral calcinosis at this time.

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No specialists are currently listed for Familial normophosphatemic tumoral calcinosis.

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 Familial normophosphatemic tumoral calcinosis.

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Community

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Caregiver Resources

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Social Security Disability

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Questions for your doctor

Bring these to your next appointment

  • Q1.How can we tell if this is normophosphatemic tumoral calcinosis versus other types?,Should my family members be tested for the SAMD9 gene mutation?,When is surgery recommended, and how likely are the deposits to come back?,Are there any medications that might slow down the formation of new calcium deposits?,What physical therapy or exercises can help maintain joint movement?,How should we care for wounds if a deposit breaks through the skin?,Are there any clinical trials or new treatments being studied for this condition?

Common questions about Familial normophosphatemic tumoral calcinosis

What is Familial normophosphatemic tumoral calcinosis?

Familial normophosphatemic tumoral calcinosis is a rare inherited condition in which large deposits of calcium form in the soft tissues of the body, especially around joints. Unlike other forms of tumoral calcinosis, the phosphate levels in the blood remain normal (hence the term 'normophosphatemic'). The calcium deposits, sometimes called calcinotic masses, tend to appear around the hips, shoulders, elbows, knees, and other joints. These masses can grow quite large and may cause pain, limit movement, and sometimes break through the skin, leading to drainage of a chalky white material. The con

How is Familial normophosphatemic tumoral calcinosis inherited?

Familial normophosphatemic tumoral calcinosis follows a autosomal recessive inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.

At what age does Familial normophosphatemic tumoral calcinosis typically begin?

Typical onset of Familial normophosphatemic tumoral calcinosis is childhood. Age of onset can vary across affected individuals.