CLCN4-related X-linked intellectual disability syndrome

Last reviewed

🖨 Print for my doctorAdvocacy Hub →
ORPHA:485350OMIM:300114F78.1
Who is this for?
Show terms as
8Treatment centers

Where are you in your journey?

UniteRare data is sourced from FDA.gov, ClinicalTrials.gov, Orphanet, OMIM, and NORD.
Report missing data

Overview

CLCN4-related X-linked intellectual disability syndrome is a rare genetic condition caused by changes (mutations) in the CLCN4 gene. This gene provides instructions for making a protein called a chloride channel, which helps control the flow of electrically charged particles in and out of brain cells. When this channel does not work properly, it affects how brain cells communicate, leading to problems with learning, behavior, and development. The condition mainly affects intellectual ability and behavior. People with this syndrome often have mild to moderate intellectual disability, meaning they may need extra support with learning and daily tasks. Many also experience behavioral challenges, seizures, and difficulties with speech and language. Some individuals have features that affect their physical appearance, though these vary widely from person to person. Because this is an X-linked condition, it can affect both males and females, though males are often more severely affected. Females who carry one changed copy of the gene may have milder symptoms or, in some cases, significant difficulties as well. There is currently no cure, but supportive therapies such as speech therapy, occupational therapy, and seizure medications can help manage symptoms and improve quality of life.

Also known as:

Key symptoms:

Intellectual disability (mild to moderate difficulty with learning and thinking)Delayed speech and language developmentBehavioral problems such as aggression, anxiety, or hyperactivitySeizures (epilepsy)Autism-like behaviors or featuresDelayed motor milestones such as sitting or walking later than expectedAttention difficultiesMood swings or emotional dysregulationMild differences in facial featuresSleep problems

Clinical phenotype terms (45)— hover any for plain English
Inheritance

X-linked dominant

Carried on the X chromosome; a single copy can cause the condition

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 CLCN4-related X-linked intellectual disability syndrome.

View clinical trials →

No actively recruiting trials found for CLCN4-related X-linked intellectual disability syndrome at this time.

New trials open frequently. Follow this disease to get notified.

Search ClinicalTrials.gov ↗Join the CLCN4-related X-linked intellectual disability syndrome community →

No specialists are currently listed for CLCN4-related X-linked intellectual disability 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 CLCN4-related X-linked intellectual disability syndrome.

Search all travel grants →NORD Financial Assistance ↗

Community

Open CLCN4-related X-linked intellectual disability syndromeForum →

No community posts yet. Be the first to share your experience with CLCN4-related X-linked intellectual disability syndrome.

Start the conversation →

Latest news about CLCN4-related X-linked intellectual disability syndrome

No recent news articles for CLCN4-related X-linked intellectual disability syndrome.

Follow this condition to be notified when news becomes available.

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 type of genetic change does my child have in the CLCN4 gene, and what does that mean for their prognosis?,Should other family members be tested for this condition?,What therapies do you recommend, and how soon should we start?,What seizure medications are most appropriate, and what are the side effects to watch for?,Are there any clinical trials or research studies we could participate in?,What school supports or educational plans should we ask for?,What signs should prompt us to seek emergency care?

Common questions about CLCN4-related X-linked intellectual disability syndrome

What is CLCN4-related X-linked intellectual disability syndrome?

CLCN4-related X-linked intellectual disability syndrome is a rare genetic condition caused by changes (mutations) in the CLCN4 gene. This gene provides instructions for making a protein called a chloride channel, which helps control the flow of electrically charged particles in and out of brain cells. When this channel does not work properly, it affects how brain cells communicate, leading to problems with learning, behavior, and development. The condition mainly affects intellectual ability and behavior. People with this syndrome often have mild to moderate intellectual disability, meaning t

How is CLCN4-related X-linked intellectual disability syndrome inherited?

CLCN4-related X-linked intellectual disability syndrome follows a x-linked dominant inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.

At what age does CLCN4-related X-linked intellectual disability syndrome typically begin?

Typical onset of CLCN4-related X-linked intellectual disability syndrome is infantile. Age of onset can vary across affected individuals.