Isolated exencephaly

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Overview

Isolated exencephaly is a very rare and severe birth defect of the brain and skull. In this condition, the brain develops outside of the skull instead of being protected inside it. The skull bones do not close properly during early pregnancy, leaving the brain exposed. This is different from anencephaly, where much of the brain is absent — in exencephaly, the brain tissue is present but sits outside the skull, often covered only by a thin membrane or exposed directly. The term 'isolated' means this condition occurs on its own, without other major birth defects being the main feature. Exencephaly is considered a neural tube defect, meaning it happens when the neural tube — the structure that becomes the brain and spinal cord — does not close correctly in the first few weeks of pregnancy. This usually occurs between 3 and 4 weeks after conception, often before a person even knows they are pregnant. Because the brain is unprotected and exposed, this condition is not compatible with life outside the womb in most cases. Babies diagnosed with exencephaly typically do not survive to birth or pass away very shortly after. There is no cure or treatment that can repair the brain or skull in this condition. Care is focused on supporting families through the diagnosis and, in some cases, comfort care for the newborn.

Key symptoms:

Brain tissue visible outside the skull at birthAbsent or incomplete skull bones over the top of the headAbnormal head shapeExposed brain tissue, sometimes covered by a thin membraneNo normal scalp covering the top of the headStillbirth or death shortly after birth in most casesAbnormal fetal movements detected during pregnancy in some cases

Clinical phenotype terms (16)— hover any for plain English
Abnormal calvaria morphologyHP:0002683Hypoplasia of the frontal boneHP:0005466Maternal diabetesHP:0009800Anterior pituitary hypoplasiaHP:0010627Posterior pituitary agenesisHP:0011756Abnormal facial skeleton morphologyHP:0011821Maternal fever in pregnancyHP:0030244
Inheritance

Multifactorial

Caused by a mix of several genes and environmental factors

Age of Onset

Neonatal

Begins at or shortly after birth (first 4 weeks)

Orphanet ↗NORD ↗

Treatments

No FDA-approved treatments are currently listed for Isolated exencephaly.

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No actively recruiting trials found for Isolated exencephaly at this time.

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No specialists are currently listed for Isolated exencephaly.

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 Isolated exencephaly.

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Community

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Caregiver Resources

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Learn how rare disease patients may qualify for SSDI/SSI benefits.

Questions for your doctor

Bring these to your next appointment

  • Q1.What caused this to happen, and is there a genetic reason we should know about?,What is the chance this could happen again in a future pregnancy?,What dose of folic acid should I take before and during my next pregnancy?,What are our options now that we have this diagnosis?,What does comfort care look like if our baby is born alive?,Are there support groups or counselors who specialize in helping families through this kind of loss?,Should we have genetic testing done, and if so, what type?

Common questions about Isolated exencephaly

What is Isolated exencephaly?

Isolated exencephaly is a very rare and severe birth defect of the brain and skull. In this condition, the brain develops outside of the skull instead of being protected inside it. The skull bones do not close properly during early pregnancy, leaving the brain exposed. This is different from anencephaly, where much of the brain is absent — in exencephaly, the brain tissue is present but sits outside the skull, often covered only by a thin membrane or exposed directly. The term 'isolated' means this condition occurs on its own, without other major birth defects being the main feature. Exenceph

How is Isolated exencephaly inherited?

Isolated exencephaly follows a multifactorial inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.

At what age does Isolated exencephaly typically begin?

Typical onset of Isolated exencephaly is neonatal. Age of onset can vary across affected individuals.