CTCF-related neurodevelopmental disorder

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ORPHA:363611OMIM:615502Q87.8
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Overview

CTCF-related neurodevelopmental disorder is a rare genetic condition caused by changes (mutations) in the CTCF gene. This gene provides instructions for making a protein that plays a critical role in how other genes are turned on and off during development, especially brain development. Because this protein is so important for regulating many other genes, changes in CTCF can affect multiple body systems. Children with this condition typically show intellectual disability that ranges from mild to moderate, along with delays in reaching developmental milestones such as sitting, walking, and talking. Many affected individuals have distinctive facial features, which may include a broad forehead, widely spaced eyes, a flat nasal bridge, and a thin upper lip. Feeding difficulties in infancy are common, and some children have short stature or low birth weight. Behavioral challenges, including features of autism spectrum disorder and attention difficulties, are also frequently reported. There is currently no cure for CTCF-related neurodevelopmental disorder. Treatment focuses on managing symptoms and supporting development through therapies such as speech therapy, occupational therapy, and physical therapy. Early intervention services can make a meaningful difference in a child's progress. The condition is also sometimes referred to as intellectual disability autosomal dominant 21 (MRD21) or CTCF-related disorder.

Key symptoms:

Intellectual disability (mild to moderate)Delayed speech and language developmentDelayed motor milestones like walkingFeeding difficulties in infancyShort stature or poor growthLow birth weightSmall head size (microcephaly)Distinctive facial featuresBehavioral challengesFeatures of autism spectrum disorderAttention difficultiesLearning difficultiesLow muscle tone (floppiness)Heart defects in some casesVision or eye problems

Clinical phenotype terms (50)— hover any for plain English
Macrodontia of permanent maxillary central incisorHP:0000675
Inheritance

Autosomal dominant

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

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 CTCF-related neurodevelopmental disorder.

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No actively recruiting trials found for CTCF-related neurodevelopmental disorder at this time.

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Search ClinicalTrials.gov ↗Join the CTCF-related neurodevelopmental disorder community →

No specialists are currently listed for CTCF-related neurodevelopmental disorder.

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 CTCF-related neurodevelopmental disorder.

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Community

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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.What specific CTCF gene change does my child have, and what does it mean for their development?,What therapies should we start right away, and how often should they occur?,Does my child need a heart evaluation or any other organ screening?,What educational supports or accommodations should we request at school?,Is there a risk of this condition occurring again in future pregnancies?,Are there any clinical studies or research programs we could participate in?,What specialists should we see regularly, and how often?

Common questions about CTCF-related neurodevelopmental disorder

What is CTCF-related neurodevelopmental disorder?

CTCF-related neurodevelopmental disorder is a rare genetic condition caused by changes (mutations) in the CTCF gene. This gene provides instructions for making a protein that plays a critical role in how other genes are turned on and off during development, especially brain development. Because this protein is so important for regulating many other genes, changes in CTCF can affect multiple body systems. Children with this condition typically show intellectual disability that ranges from mild to moderate, along with delays in reaching developmental milestones such as sitting, walking, and tal

How is CTCF-related neurodevelopmental disorder inherited?

CTCF-related neurodevelopmental disorder follows a autosomal dominant inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.

At what age does CTCF-related neurodevelopmental disorder typically begin?

Typical onset of CTCF-related neurodevelopmental disorder is infantile. Age of onset can vary across affected individuals.