Intellectual disability-seizures-macrocephaly-obesity syndrome

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Overview

Intellectual disability-seizures-macrocephaly-obesity syndrome is an extremely rare genetic condition that affects brain development and body growth. It is sometimes referred to by its associated gene name, MAST1-related syndrome. People with this condition typically have intellectual disability, which means they may have difficulty learning, reasoning, and problem-solving compared to others their age. Seizures (episodes of abnormal electrical activity in the brain) are another hallmark feature and can vary in type and severity. Macrocephaly, meaning a head circumference that is larger than expected, is often noticed early in life. Obesity or significant weight gain tends to develop during childhood. The syndrome may also include delays in reaching developmental milestones such as sitting, walking, and talking. Some individuals may have behavioral challenges. Because this is a very recently described and rare condition, the full range of symptoms is still being understood. There is currently no cure for this syndrome. Treatment focuses on managing individual symptoms, such as anti-seizure medications for epilepsy, educational support for intellectual disability, dietary and lifestyle interventions for obesity, and therapies like speech and occupational therapy to support development. Early intervention and a coordinated care team can help improve quality of life.

Also known as:

Key symptoms:

Intellectual disability (difficulty with learning and reasoning)Seizures or epilepsyLarger than normal head size (macrocephaly)Obesity or excessive weight gainDelayed speech and language developmentDelayed motor milestones (sitting, walking)Behavioral difficultiesLow muscle tone (hypotonia)Feeding difficulties in infancyPossible abnormalities on brain MRI

Clinical phenotype terms (50)— hover any for plain English
Periorbital fullnessHP:0000629
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 ↗NORD ↗

Treatments

No FDA-approved treatments are currently listed for Intellectual disability-seizures-macrocephaly-obesity syndrome.

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No actively recruiting trials found for Intellectual disability-seizures-macrocephaly-obesity syndrome at this time.

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Search ClinicalTrials.gov ↗Join the Intellectual disability-seizures-macrocephaly-obesity syndrome community →

No specialists are currently listed for Intellectual disability-seizures-macrocephaly-obesity 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 Intellectual disability-seizures-macrocephaly-obesity syndrome.

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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

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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.What type of seizures does my child have, and what is the best medication to control them?,Are there any clinical trials or research studies we could participate in?,What therapies (speech, occupational, physical) should we start, and how often?,How should we manage my child's weight, and should we see a dietitian?,What should I do if my child has a prolonged seizure at home?,How often should we have follow-up brain imaging or EEG testing?,What educational supports and accommodations should we request at school?

Common questions about Intellectual disability-seizures-macrocephaly-obesity syndrome

What is Intellectual disability-seizures-macrocephaly-obesity syndrome?

Intellectual disability-seizures-macrocephaly-obesity syndrome is an extremely rare genetic condition that affects brain development and body growth. It is sometimes referred to by its associated gene name, MAST1-related syndrome. People with this condition typically have intellectual disability, which means they may have difficulty learning, reasoning, and problem-solving compared to others their age. Seizures (episodes of abnormal electrical activity in the brain) are another hallmark feature and can vary in type and severity. Macrocephaly, meaning a head circumference that is larger than ex

How is Intellectual disability-seizures-macrocephaly-obesity syndrome inherited?

Intellectual disability-seizures-macrocephaly-obesity syndrome follows a autosomal dominant inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.

At what age does Intellectual disability-seizures-macrocephaly-obesity syndrome typically begin?

Typical onset of Intellectual disability-seizures-macrocephaly-obesity syndrome is infantile. Age of onset can vary across affected individuals.