Focal epilepsy-intellectual disability-cerebro-cerebellar malformation

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ORPHA:352587OMIM:605021Q04.8
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Overview

Focal epilepsy-intellectual disability-cerebro-cerebellar malformation is an extremely rare genetic condition that affects brain development and function. People with this condition are born with structural abnormalities in both the cerebrum (the main part of the brain responsible for thinking and movement) and the cerebellum (the part of the brain that helps with balance and coordination). These brain malformations lead to seizures that start in a specific area of the brain (called focal epilepsy), as well as intellectual disability that can range from mild to severe. The seizures in this condition often begin in infancy or early childhood and may be difficult to control with standard anti-seizure medications. Children with this condition typically show delays in reaching developmental milestones such as sitting, walking, and talking. The intellectual disability can affect learning, communication, and the ability to perform everyday tasks independently. There is currently no cure for this condition. Treatment focuses on managing symptoms, particularly controlling seizures with anti-epileptic medications, and providing supportive therapies such as physical therapy, occupational therapy, and speech therapy to help maximize each person's abilities and quality of life. A team of specialists is usually needed to address the various aspects of this complex condition.

Also known as:

Key symptoms:

Seizures that start in one area of the brain (focal seizures)Intellectual disabilityDelayed development in childhoodAbnormal brain structure visible on imagingAbnormal cerebellum structureProblems with balance and coordinationDelayed speech and language developmentDelayed motor milestones like sitting and walkingDifficulty with learningLow muscle toneBehavioral difficultiesProblems with fine motor skills

Inheritance

Autosomal recessive

Passed on when both parents carry the same gene change; often skips generations

Age of Onset

Infantile

Begins in infancy, roughly 1 month to 2 years old

Orphanet ↗OMIM ↗NORD ↗

Treatments

No FDA-approved treatments are currently listed for Focal epilepsy-intellectual disability-cerebro-cerebellar malformation.

View clinical trials →

No actively recruiting trials found for Focal epilepsy-intellectual disability-cerebro-cerebellar malformation at this time.

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Search ClinicalTrials.gov ↗Join the Focal epilepsy-intellectual disability-cerebro-cerebellar malformation community →

No specialists are currently listed for Focal epilepsy-intellectual disability-cerebro-cerebellar malformation.

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 Focal epilepsy-intellectual disability-cerebro-cerebellar malformation.

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Community

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Latest news about Focal epilepsy-intellectual disability-cerebro-cerebellar malformation

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Caregiver Resources

NORD Caregiver Resources

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

Mental Health Support

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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 type of seizures does my child have, and what is the best medication to control them?,Should we pursue genetic testing, and what can the results tell us about prognosis?,What therapies (physical, occupational, speech) do you recommend, and how often?,What should I do if my child has a prolonged seizure or a seizure that looks different from usual?,Are there any clinical trials or new treatments being studied for this condition?,What educational supports or accommodations should we request at school?,How often should we schedule follow-up brain imaging and EEG monitoring?

Common questions about Focal epilepsy-intellectual disability-cerebro-cerebellar malformation

What is Focal epilepsy-intellectual disability-cerebro-cerebellar malformation?

Focal epilepsy-intellectual disability-cerebro-cerebellar malformation is an extremely rare genetic condition that affects brain development and function. People with this condition are born with structural abnormalities in both the cerebrum (the main part of the brain responsible for thinking and movement) and the cerebellum (the part of the brain that helps with balance and coordination). These brain malformations lead to seizures that start in a specific area of the brain (called focal epilepsy), as well as intellectual disability that can range from mild to severe. The seizures in this co

How is Focal epilepsy-intellectual disability-cerebro-cerebellar malformation inherited?

Focal epilepsy-intellectual disability-cerebro-cerebellar malformation follows a autosomal recessive inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.

At what age does Focal epilepsy-intellectual disability-cerebro-cerebellar malformation typically begin?

Typical onset of Focal epilepsy-intellectual disability-cerebro-cerebellar malformation is infantile. Age of onset can vary across affected individuals.