Antley-Bixler syndrome with genital anomaly and disorder of steroidogenesis

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ORPHA:63269OMIM:201750Q87.8
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Overview

Antley-Bixler syndrome with genital anomaly and disorder of steroidogenesis (ABS with steroidogenesis defect) is a rare genetic condition caused by changes in the POR gene, which provides instructions for making an enzyme called P450 oxidoreductase. This enzyme is essential for the body to produce steroid hormones — including cortisol, sex hormones, and other important chemicals — and for processing certain medications. When POR does not work properly, the body cannot make these hormones correctly, leading to a wide range of problems. The condition affects the skeleton, the hormone system, and the development of reproductive organs. Babies are often born with fused skull bones (craniosynostosis), which can affect the shape of the head and face. The bones of the arms and legs may also be unusually shaped or bent. Because steroid hormone production is disrupted, both boys and girls may have ambiguous or atypical genitalia at birth, meaning it can be hard to tell the sex of the baby from appearance alone. Girls may show signs of masculinization, while boys may appear undermasculinized. Treatment focuses on managing the individual symptoms. Surgery may be needed to correct skull or limb problems. Hormone replacement therapy can help address the steroid deficiency, particularly cortisol, which is critical for handling stress and illness. With careful medical management, many children can do well, though lifelong monitoring is usually needed.

Also known as:

Key symptoms:

Fused skull bones (craniosynostosis) causing an unusual head shapeMidface underdevelopment giving a flattened facial appearanceBowed or bent arm and leg bonesStiff or fused joints (joint contractures) limiting movementAmbiguous or atypical genitalia at birthLow cortisol levels (adrenal insufficiency) causing fatigue and poor stress responseAbnormal sex hormone levels affecting puberty and fertilityChoanal stenosis or atresia (narrowed or blocked nasal passages)Ear abnormalitiesShort statureKidney or urinary tract abnormalities in some casesDelayed puberty or absent puberty without treatment

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 Antley-Bixler syndrome with genital anomaly and disorder of steroidogenesis.

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No actively recruiting trials found for Antley-Bixler syndrome with genital anomaly and disorder of steroidogenesis at this time.

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No specialists are currently listed for Antley-Bixler syndrome with genital anomaly and disorder of steroidogenesis.

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 Antley-Bixler syndrome with genital anomaly and disorder of steroidogenesis.

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Community

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Caregiver Resources

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Questions for your doctor

Bring these to your next appointment

  • Q1.Does my child need cortisol replacement therapy, and how do I manage sick-day dosing at home?,What surgeries might be needed for the skull or limbs, and when should they happen?,How will my child's hormone levels be monitored as they grow, and what should I watch for at puberty?,What is the plan for managing my child's genital development, and who should be involved in those decisions?,Are there any restrictions on activities or medications because of the adrenal insufficiency?,Should other family members be tested for the POR gene change?,What support services — such as physiotherapy, psychology, or educational support — should we be accessing?

Common questions about Antley-Bixler syndrome with genital anomaly and disorder of steroidogenesis

What is Antley-Bixler syndrome with genital anomaly and disorder of steroidogenesis?

Antley-Bixler syndrome with genital anomaly and disorder of steroidogenesis (ABS with steroidogenesis defect) is a rare genetic condition caused by changes in the POR gene, which provides instructions for making an enzyme called P450 oxidoreductase. This enzyme is essential for the body to produce steroid hormones — including cortisol, sex hormones, and other important chemicals — and for processing certain medications. When POR does not work properly, the body cannot make these hormones correctly, leading to a wide range of problems. The condition affects the skeleton, the hormone system, an

How is Antley-Bixler syndrome with genital anomaly and disorder of steroidogenesis inherited?

Antley-Bixler syndrome with genital anomaly and disorder of steroidogenesis follows a autosomal recessive inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.

At what age does Antley-Bixler syndrome with genital anomaly and disorder of steroidogenesis typically begin?

Typical onset of Antley-Bixler syndrome with genital anomaly and disorder of steroidogenesis is neonatal. Age of onset can vary across affected individuals.