Familial schizencephaly

Last reviewed

🖨 Print for my doctorAdvocacy Hub →
ORPHA:481986OMIM:269160Q04.6
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

Familial schizencephaly is a rare brain development disorder where abnormal clefts or splits form in the brain tissue during fetal development. These clefts are lined with gray matter (the thinking part of the brain) and can appear on one side of the brain (unilateral) or both sides (bilateral). The condition is called 'familial' because it runs in families, meaning it is passed down through genes, which sets it apart from the more common sporadic (non-inherited) form of schizencephaly. The clefts in the brain can disrupt normal brain function in many ways. People with this condition may experience seizures, muscle weakness or stiffness, problems with movement and coordination, and delays in reaching developmental milestones like talking or walking. The severity of symptoms depends largely on the size and location of the clefts and whether one or both sides of the brain are affected. Bilateral (both-sided) clefts tend to cause more significant challenges than unilateral (one-sided) clefts. There is currently no cure for familial schizencephaly. Treatment focuses on managing symptoms and improving quality of life. This may include anti-seizure medications, physical therapy, occupational therapy, and speech therapy. Early intervention and a strong support team can make a meaningful difference in helping individuals reach their full potential.

Key symptoms:

Seizures or epilepsyMuscle stiffness or spasticityMuscle weakness on one or both sides of the bodyDelayed speech and language developmentDelayed motor milestones such as sitting, crawling, or walkingIntellectual disability (ranges from mild to severe)Difficulty with coordination and balanceParalysis or weakness on one side of the body (hemiplegia)Vision problemsSmall head size (microcephaly) in some casesFeeding difficulties in infancy

Inheritance

Variable

Can be inherited in different ways depending on the underlying gene

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 Familial schizencephaly.

View clinical trials →

No actively recruiting trials found for Familial schizencephaly at this time.

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

Search ClinicalTrials.gov ↗Join the Familial schizencephaly community →

No specialists are currently listed for Familial schizencephaly.

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 Familial schizencephaly.

Search all travel grants →NORD Financial Assistance ↗

Community

Open Familial schizencephalyForum →

No community posts yet. Be the first to share your experience with Familial schizencephaly.

Start the conversation →

Latest news about Familial schizencephaly

No recent news articles for Familial schizencephaly.

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.Which genes should be tested in our family, and what does a positive or negative result mean for us?,What type and size of cleft does my child have, and how does that affect their prognosis?,What is the best anti-seizure medication for my child's type of epilepsy, and what are the side effects?,What therapies should we start right away, and how often should they happen?,Are there any clinical trials or research studies we could participate in?,What should we do if my child has a prolonged seizure at home?,Should other family members be tested or screened for this condition?

Common questions about Familial schizencephaly

What is Familial schizencephaly?

Familial schizencephaly is a rare brain development disorder where abnormal clefts or splits form in the brain tissue during fetal development. These clefts are lined with gray matter (the thinking part of the brain) and can appear on one side of the brain (unilateral) or both sides (bilateral). The condition is called 'familial' because it runs in families, meaning it is passed down through genes, which sets it apart from the more common sporadic (non-inherited) form of schizencephaly. The clefts in the brain can disrupt normal brain function in many ways. People with this condition may expe

At what age does Familial schizencephaly typically begin?

Typical onset of Familial schizencephaly is neonatal. Age of onset can vary across affected individuals.