Polymicrogyria due to TUBB2B mutation

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ORPHA:300573OMIM:610031Q04.3
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Overview

Polymicrogyria due to TUBB2B mutation is a rare brain development disorder that affects how the surface of the brain forms before birth. The brain normally has a pattern of folds and grooves, but in this condition, the brain develops too many small folds (called polymicrogyria), which disrupts normal brain function. This happens because of a change (mutation) in the TUBB2B gene, which provides instructions for making a protein called beta-tubulin, essential for building the internal scaffolding of brain cells during early development. When this protein doesn't work properly, brain cells cannot migrate to their correct positions, leading to the abnormal folding pattern. Symptoms vary widely but often include intellectual disability, seizures (epilepsy), delayed motor development, and difficulties with speech and language. Some children may also have problems with eye movements, feeding difficulties, and muscle stiffness or weakness. The severity depends on how much of the brain is affected — some individuals have polymicrogyria limited to certain brain regions, while others have more widespread involvement. There is currently no cure for this condition. Treatment focuses on managing symptoms, particularly controlling seizures with anti-epileptic medications, and supporting development through physical therapy, occupational therapy, and speech therapy. Early intervention programs can help children reach their best potential. A team of specialists typically works together to provide comprehensive care throughout the person's life.

Key symptoms:

Seizures or epilepsyIntellectual disabilityDelayed motor milestones such as sitting and walkingSpeech and language delaysMuscle stiffness or spasticityDifficulty with fine motor skillsFeeding difficulties in infancyAbnormal eye movementsProblems with balance and coordinationMicrocephaly (smaller than average head size)Learning difficultiesDifficulty swallowingWeakness on one or both sides of the body

Clinical phenotype terms (30)— hover any for plain English
Cortical dysplasiaHP:0002539Gray matter heterotopiaHP:0002282Abnormal caudate nucleus morphologyHP:0002339Abnormal brainstem morphologyHP:0002363Cavum septum pellucidumHP:0002389Lateral ventricle dilatationHP:0006956Oromotor apraxiaHP:0007301
Inheritance

Autosomal dominant

Passed on from just one parent; each child has about a 50% chance of inheriting it

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 Polymicrogyria due to TUBB2B mutation.

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No actively recruiting trials found for Polymicrogyria due to TUBB2B mutation at this time.

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Search ClinicalTrials.gov ↗Join the Polymicrogyria due to TUBB2B mutation community →

No specialists are currently listed for Polymicrogyria due to TUBB2B mutation.

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 Polymicrogyria due to TUBB2B mutation.

Search all travel grants →NORD Financial Assistance ↗

Community

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Latest news about Polymicrogyria due to TUBB2B mutation

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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.How much of my child's brain is affected by polymicrogyria, and what does that mean for their development?,What seizure medications do you recommend, and what side effects should I watch for?,Should we pursue genetic testing for other family members, and what is the chance of this happening in a future pregnancy?,What therapies should we start right away, and how often should they occur?,Are there any clinical trials or emerging treatments that might be relevant for my child?,What is the long-term outlook for my child's motor skills, speech, and learning?,When should I call 911 or go to the emergency room for a seizure, and should we have rescue medication at home?

Common questions about Polymicrogyria due to TUBB2B mutation

What is Polymicrogyria due to TUBB2B mutation?

Polymicrogyria due to TUBB2B mutation is a rare brain development disorder that affects how the surface of the brain forms before birth. The brain normally has a pattern of folds and grooves, but in this condition, the brain develops too many small folds (called polymicrogyria), which disrupts normal brain function. This happens because of a change (mutation) in the TUBB2B gene, which provides instructions for making a protein called beta-tubulin, essential for building the internal scaffolding of brain cells during early development. When this protein doesn't work properly, brain cells cannot

How is Polymicrogyria due to TUBB2B mutation inherited?

Polymicrogyria due to TUBB2B mutation follows a autosomal dominant inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.

At what age does Polymicrogyria due to TUBB2B mutation typically begin?

Typical onset of Polymicrogyria due to TUBB2B mutation is neonatal. Age of onset can vary across affected individuals.