Cerebrofaciothoracic dysplasia

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ORPHA:1394OMIM:616994Q87.5
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Overview

Cerebrofaciothoracic dysplasia, also known as Pascual-Castroviejo syndrome, is an extremely rare genetic condition that affects the development of the brain, face, and chest (thorax). People with this condition typically have distinctive facial features, abnormalities of the rib cage and spine, and varying degrees of intellectual disability. The facial features may include a broad forehead, widely spaced eyes, a flat nasal bridge, and a small jaw. Chest abnormalities often involve short or fused ribs and an unusually shaped rib cage, which can sometimes affect breathing. Vertebral (spine) abnormalities are also common and may include fused or malformed vertebrae. Some individuals may also have short stature and limb abnormalities. Because this condition is so rare, with only a handful of cases described in the medical literature, the full range of symptoms and the best approaches to treatment are still being understood. There is currently no cure for cerebrofaciothoracic dysplasia. Treatment focuses on managing individual symptoms, which may include surgical correction of skeletal abnormalities, respiratory support if breathing is affected, and educational support for developmental delays. A team of specialists is usually needed to provide comprehensive care.

Also known as:

Key symptoms:

Distinctive facial features such as broad forehead and widely spaced eyesSmall or receding jawFlat nasal bridgeShort or fused ribsAbnormally shaped chestFused or malformed vertebrae in the spineShort statureIntellectual disability or developmental delaysBreathing difficulties due to small chestLimb abnormalitiesLow-set earsDelayed speech development

Clinical phenotype terms (35)— hover any for plain English
Broad philtrumHP:0000289Bifid ribsHP:0000892Rib fusionHP:0000902HemivertebraeHP:0002937
Inheritance

Autosomal recessive

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

Age of Onset

Neonatal

Begins at or shortly after birth (first 4 weeks)

Orphanet ↗OMIM ↗NORD ↗

Treatments

No FDA-approved treatments are currently listed for Cerebrofaciothoracic dysplasia.

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No actively recruiting trials found for Cerebrofaciothoracic dysplasia at this time.

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No specialists are currently listed for Cerebrofaciothoracic dysplasia.

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 Cerebrofaciothoracic dysplasia.

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Community

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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 severe are my child's chest and spine abnormalities, and will they need surgery?,What breathing support might my child need now or in the future?,What developmental therapies should we start, and how often?,Should we pursue genetic testing, and what might it tell us?,What is the expected developmental outlook for my child?,Are there any clinical studies or research programs we could participate in?,What specialists should be part of my child's care team?

Common questions about Cerebrofaciothoracic dysplasia

What is Cerebrofaciothoracic dysplasia?

Cerebrofaciothoracic dysplasia, also known as Pascual-Castroviejo syndrome, is an extremely rare genetic condition that affects the development of the brain, face, and chest (thorax). People with this condition typically have distinctive facial features, abnormalities of the rib cage and spine, and varying degrees of intellectual disability. The facial features may include a broad forehead, widely spaced eyes, a flat nasal bridge, and a small jaw. Chest abnormalities often involve short or fused ribs and an unusually shaped rib cage, which can sometimes affect breathing. Vertebral (spine) abno

How is Cerebrofaciothoracic dysplasia inherited?

Cerebrofaciothoracic dysplasia follows a autosomal recessive inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.

At what age does Cerebrofaciothoracic dysplasia typically begin?

Typical onset of Cerebrofaciothoracic dysplasia is neonatal. Age of onset can vary across affected individuals.