Aprosencephaly/atelencephaly spectrum

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Overview

Aprosencephaly/atelencephaly spectrum is a very rare and severe brain development condition that is present before birth. In this condition, the front part of the brain — called the prosencephalon or telencephalon — either fails to form at all or forms in a very incomplete way. The brain is one of the most important organs in the body, and when its front sections do not develop properly, it affects nearly every function the body controls, including movement, thinking, breathing, and basic survival. Babies born with this condition typically have a very small or absent forebrain, which means the brain cannot send or receive the signals needed to support normal life. This is different from other brain malformation conditions like holoprosencephaly, where the brain forms but does not divide correctly. In aprosencephaly and atelencephaly, the brain tissue itself is largely missing from the start. Because this condition is so severe, most affected pregnancies end in miscarriage or stillbirth. Babies who are born alive typically survive only for a very short time after birth. There is currently no cure or treatment that can repair the missing brain tissue. Care is focused on keeping the baby comfortable and supporting the family through this very difficult experience. Families are encouraged to work closely with a team of specialists and genetic counselors to understand what happened and what it might mean for future pregnancies.

Also known as:

Key symptoms:

Absent or severely underdeveloped forebrainVery small head size (microcephaly) or abnormal skull shapeAbsence of normal facial features in some casesInability to breathe independentlyNo response to light, sound, or touchAbsence of normal brain activitySeizures in some casesInability to feed or swallowAbnormal or absent reflexesStillbirth or death shortly after birth in most cases

Inheritance

Sporadic

Usually appears on its own, not inherited from a parent

Age of Onset

Neonatal

Begins at or shortly after birth (first 4 weeks)

Orphanet ↗NORD ↗

Treatments

No FDA-approved treatments are currently listed for Aprosencephaly/atelencephaly spectrum.

View clinical trials →

No actively recruiting trials found for Aprosencephaly/atelencephaly spectrum at this time.

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

Search ClinicalTrials.gov ↗Join the Aprosencephaly/atelencephaly spectrum community →

No specialists are currently listed for Aprosencephaly/atelencephaly spectrum.

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 Aprosencephaly/atelencephaly spectrum.

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Community

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Caregiver Resources

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

Mental Health Support

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Family & Caregiver Grants

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Social Security Disability

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Questions for your doctor

Bring these to your next appointment

  • Q1.What exactly is missing or abnormal in my baby's brain, and what does that mean for survival?,Are there any genetic tests we should do to understand why this happened?,What are the chances this could happen again in a future pregnancy?,What does comfort care look like for a baby born with this condition?,Can we meet with a palliative care team before the birth to make a plan?,Are there support groups or counselors who specialize in helping families going through this?,Should we consider an autopsy after birth, and what information could it give us?

Common questions about Aprosencephaly/atelencephaly spectrum

What is Aprosencephaly/atelencephaly spectrum?

Aprosencephaly/atelencephaly spectrum is a very rare and severe brain development condition that is present before birth. In this condition, the front part of the brain — called the prosencephalon or telencephalon — either fails to form at all or forms in a very incomplete way. The brain is one of the most important organs in the body, and when its front sections do not develop properly, it affects nearly every function the body controls, including movement, thinking, breathing, and basic survival. Babies born with this condition typically have a very small or absent forebrain, which means th

How is Aprosencephaly/atelencephaly spectrum inherited?

Aprosencephaly/atelencephaly spectrum follows a sporadic inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.

At what age does Aprosencephaly/atelencephaly spectrum typically begin?

Typical onset of Aprosencephaly/atelencephaly spectrum is neonatal. Age of onset can vary across affected individuals.