Early-onset epileptic encephalopathy-cortical blindness-intellectual disability-facial dysmorphism syndrome

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ORPHA:411986OMIM:615859G40.4
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Overview

Early-onset epileptic encephalopathy-cortical blindness-intellectual disability-facial dysmorphism syndrome is an extremely rare genetic condition that affects the brain and its development from very early in life. The name describes its main features: seizures that begin in infancy or early childhood (epileptic encephalopathy), vision loss caused by problems in the brain's visual processing areas rather than the eyes themselves (cortical blindness), significant challenges with thinking and learning (intellectual disability), and distinctive facial features (facial dysmorphism). The seizures in this condition are often severe and difficult to control with standard anti-seizure medications. Because the brain is affected so broadly, children with this syndrome typically experience major delays in reaching developmental milestones such as sitting, walking, and speaking. The cortical blindness means the eyes may appear normal on examination, but the brain cannot properly process what the eyes see. Facial features may include subtle differences in the shape of the face, eyes, nose, or ears that a trained geneticist can recognize. Currently, there is no cure for this condition. Treatment focuses on managing seizures with anti-epileptic drugs, providing supportive therapies such as physical therapy, occupational therapy, and speech therapy, and addressing vision-related needs. A multidisciplinary team of specialists is essential for providing the best possible care and quality of life.

Also known as:

Key symptoms:

Severe seizures starting in infancyVision loss due to brain problems (cortical blindness)Intellectual disabilityDistinctive facial featuresSevere developmental delaysDifficulty or inability to walkDifficulty or inability to speakLow muscle tone (floppiness)Feeding difficultiesSmall head size (microcephaly)Abnormal movementsPoor response to visual stimuliLimited ability to interact with surroundings

Clinical phenotype terms (36)— hover any for plain English
Periorbital fullnessHP:0000629Generalized tonic seizureHP:0010818Multifocal epileptiform dischargesHP:0010841Occipital cortical atrophyHP:0012105Hypoplasia of the ponsHP:0012110
Inheritance

Autosomal recessive

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

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 Early-onset epileptic encephalopathy-cortical blindness-intellectual disability-facial dysmorphism syndrome.

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No actively recruiting trials found for Early-onset epileptic encephalopathy-cortical blindness-intellectual disability-facial dysmorphism syndrome at this time.

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No specialists are currently listed for Early-onset epileptic encephalopathy-cortical blindness-intellectual disability-facial dysmorphism syndrome.

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 Early-onset epileptic encephalopathy-cortical blindness-intellectual disability-facial dysmorphism syndrome.

Search all travel grants →NORD Financial Assistance ↗

Community

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Caregiver Resources

NORD Caregiver Resources

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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 plan to control them?,Should we consider a ketogenic diet or other non-medication approaches for seizure control?,What genetic test confirmed the diagnosis, and what does this mean for future pregnancies?,How can we best support my child's vision and overall development?,What therapies should my child be receiving, and how often?,Are there any clinical trials or research studies that my child might be eligible for?,What emergency plan should we have in place for prolonged seizures?

Common questions about Early-onset epileptic encephalopathy-cortical blindness-intellectual disability-facial dysmorphism syndrome

What is Early-onset epileptic encephalopathy-cortical blindness-intellectual disability-facial dysmorphism syndrome?

Early-onset epileptic encephalopathy-cortical blindness-intellectual disability-facial dysmorphism syndrome is an extremely rare genetic condition that affects the brain and its development from very early in life. The name describes its main features: seizures that begin in infancy or early childhood (epileptic encephalopathy), vision loss caused by problems in the brain's visual processing areas rather than the eyes themselves (cortical blindness), significant challenges with thinking and learning (intellectual disability), and distinctive facial features (facial dysmorphism). The seizures i

How is Early-onset epileptic encephalopathy-cortical blindness-intellectual disability-facial dysmorphism syndrome inherited?

Early-onset epileptic encephalopathy-cortical blindness-intellectual disability-facial dysmorphism syndrome follows a autosomal recessive inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.

At what age does Early-onset epileptic encephalopathy-cortical blindness-intellectual disability-facial dysmorphism syndrome typically begin?

Typical onset of Early-onset epileptic encephalopathy-cortical blindness-intellectual disability-facial dysmorphism syndrome is neonatal. Age of onset can vary across affected individuals.