Progressive microcephaly-seizures-cortical blindness-developmental delay syndrome

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ORPHA:477814OMIM:616632Q02
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Overview

Progressive microcephaly-seizures-cortical blindness-developmental delay syndrome is a very rare brain development disorder that affects children from birth or early infancy. The name describes the main features: the head grows more slowly than expected (microcephaly), leading to a smaller-than-normal head size over time; seizures (epilepsy) that can be hard to control; cortical blindness, which means the eyes themselves may be healthy but the brain cannot properly process what they see; and significant delays in development, including learning, movement, and communication skills. This condition is caused by changes (mutations) in genes that are important for normal brain growth and function. Because the brain does not develop properly, children with this syndrome face challenges across many areas of life. Seizures can range from mild to very severe, and developmental delays mean that children may need lifelong support with daily activities, communication, and learning. There is currently no cure for this syndrome. Treatment focuses on managing symptoms, especially controlling seizures with anti-epileptic medications, and supporting development through therapies such as physical therapy, occupational therapy, and speech therapy. A team of specialists works together to give children the best possible quality of life. Early diagnosis and early intervention are important to help children reach their full potential.

Key symptoms:

Smaller-than-normal head size that falls further behind over time (progressive microcephaly)Seizures that may be frequent and hard to controlCortical blindness — difficulty seeing even though the eyes look normalSignificant delays in reaching developmental milestones like sitting, walking, and talkingIntellectual disability ranging from moderate to severeMuscle stiffness or weakness (spasticity or hypotonia)Difficulty feeding, especially in infancyLimited or absent speechPoor coordination and balanceAbnormal eye movements or lack of visual tracking

Clinical phenotype terms (22)— hover any for plain English
Hypoplastic optic chiasmHP:0034311Combined immunodeficiencyHP:0005387Metopic synostosisHP:0011330B-cell lymphomaHP:0012191
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 Progressive microcephaly-seizures-cortical blindness-developmental delay syndrome.

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No actively recruiting trials found for Progressive microcephaly-seizures-cortical blindness-developmental delay syndrome at this time.

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No specialists are currently listed for Progressive microcephaly-seizures-cortical blindness-developmental delay 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 Progressive microcephaly-seizures-cortical blindness-developmental delay syndrome.

Search all travel grants →NORD Financial Assistance ↗

Community

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Latest news about Progressive microcephaly-seizures-cortical blindness-developmental delay syndrome

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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.Which specific gene or genes are causing my child's condition, and what does that mean for our family?,What is the best seizure medication plan for my child, and what should I do if a seizure lasts more than 5 minutes?,What therapies should my child start as soon as possible, and how often should they attend?,Are there any clinical trials or research studies that my child might be eligible for?,What support services and educational resources are available for my child and our family?,Should other family members be tested for the genetic mutation?,What signs should prompt me to seek emergency care, and what is our emergency plan?

Common questions about Progressive microcephaly-seizures-cortical blindness-developmental delay syndrome

What is Progressive microcephaly-seizures-cortical blindness-developmental delay syndrome?

Progressive microcephaly-seizures-cortical blindness-developmental delay syndrome is a very rare brain development disorder that affects children from birth or early infancy. The name describes the main features: the head grows more slowly than expected (microcephaly), leading to a smaller-than-normal head size over time; seizures (epilepsy) that can be hard to control; cortical blindness, which means the eyes themselves may be healthy but the brain cannot properly process what they see; and significant delays in development, including learning, movement, and communication skills. This condit

How is Progressive microcephaly-seizures-cortical blindness-developmental delay syndrome inherited?

Progressive microcephaly-seizures-cortical blindness-developmental delay syndrome follows a autosomal recessive inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.

At what age does Progressive microcephaly-seizures-cortical blindness-developmental delay syndrome typically begin?

Typical onset of Progressive microcephaly-seizures-cortical blindness-developmental delay syndrome is infantile. Age of onset can vary across affected individuals.