Isolated anencephaly

Last reviewed

🖨 Print for my doctorAdvocacy Hub →
ORPHA:563609Q00.0
Who is this for?
Show terms as
8Treatment centers

Where are you in your journey?

UniteRare data is sourced from FDA.gov, ClinicalTrials.gov, Orphanet, OMIM, and NORD.
Report missing data

Overview

Isolated anencephaly is a very serious birth defect where a baby is born without large parts of the brain, skull, and scalp. The word 'anencephaly' comes from Greek meaning 'without a brain.' In this condition, the neural tube — the structure that forms the brain and spinal cord early in pregnancy — does not close properly during the first few weeks after conception. As a result, the developing brain is exposed and does not form correctly. The cerebral hemispheres, which control thinking, movement, and sensation, are either absent or severely underdeveloped. The brainstem, which controls basic functions like breathing and heart rate, may be partially present. Babies with anencephaly are either stillborn or survive only a short time after birth, usually hours to days. There is no treatment that can repair or replace the missing brain tissue. The condition is detected during pregnancy through routine ultrasound or blood tests. The term 'isolated' means the condition occurs on its own, without other major birth defects or a known genetic syndrome. Anencephaly is one of the most common and most severe neural tube defects.

Key symptoms:

Absence of the top and back of the skullMissing or severely underdeveloped brain tissue (cerebral hemispheres)Exposed brain tissue at the top of the headAbnormal facial features due to skull malformationInability to see, hear, or feel in a conscious wayNo awareness or consciousnessInability to breathe independently for more than a short timeAbsence of normal reflexes in most casesStillbirth or death within hours to days of birth

Clinical phenotype terms (8)— hover any for plain English
Adrenal hypoplasiaHP:0000835Maternal diabetesHP:0009800Thymus hyperplasiaHP:0010516Maternal 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 anencephaly.

View clinical trials →

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

New trials open frequently. Follow this disease to get notified.

Search ClinicalTrials.gov ↗Join the Isolated anencephaly community →

No specialists are currently listed for Isolated anencephaly.

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 anencephaly.

Search all travel grants →NORD Financial Assistance ↗

Community

Open Isolated anencephalyForum →

No community posts yet. Be the first to share your experience with Isolated anencephaly.

Start the conversation →

Latest news about Isolated anencephaly

No recent news articles for Isolated anencephaly.

Follow this condition to be notified when news becomes available.

Caregiver Resources

NORD Caregiver Resources

Support, advocacy, and financial assistance for caregivers of rare disease patients.

Mental Health Support

Rare disease caregiving can be isolating. Connect with counseling and peer support.

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.What caused this to happen, and could anything have prevented it?,What are our options now that we have this diagnosis, and what support is available to us?,What will happen at birth, and what does comfort care look like for our baby?,What is the chance this could happen again in a future pregnancy?,Should I take a higher dose of folic acid before my next pregnancy, and when should I start?,Should we have genetic testing to look for an underlying cause?,Are there support groups or counseling services you can connect us with?

Common questions about Isolated anencephaly

What is Isolated anencephaly?

Isolated anencephaly is a very serious birth defect where a baby is born without large parts of the brain, skull, and scalp. The word 'anencephaly' comes from Greek meaning 'without a brain.' In this condition, the neural tube — the structure that forms the brain and spinal cord early in pregnancy — does not close properly during the first few weeks after conception. As a result, the developing brain is exposed and does not form correctly. The cerebral hemispheres, which control thinking, movement, and sensation, are either absent or severely underdeveloped. The brainstem, which controls basic

How is Isolated anencephaly inherited?

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

At what age does Isolated anencephaly typically begin?

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