Obesity due to SIM1 deficiency

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Overview

Obesity due to SIM1 deficiency is a rare genetic condition caused by changes (mutations) in the SIM1 gene. SIM1 stands for Single-Minded Homolog 1, and this gene plays an important role in the development and function of a part of the brain called the hypothalamus. The hypothalamus is the brain's control center for hunger, fullness, and energy balance. When the SIM1 gene does not work properly, the brain has trouble regulating appetite, which leads to severe early-onset obesity. Children with this condition typically develop an intense, hard-to-control hunger (called hyperphagia) starting in early childhood. They gain weight rapidly and can become severely obese at a young age. Some individuals may also have mild developmental delays, behavioral challenges, or features that overlap with Prader-Willi-like syndrome, though the severity varies from person to person. Currently, there is no cure for obesity due to SIM1 deficiency. Treatment focuses on managing weight through structured dietary plans, behavioral strategies to control eating, regular physical activity, and close monitoring by a team of specialists. Research into medications that target the brain's appetite pathways, such as setmelanotide (Imcivree), is ongoing and may offer hope for some patients with this type of genetic obesity. Early diagnosis through genetic testing is important so that families can access appropriate support and management strategies as soon as possible.

Key symptoms:

Severe obesity starting in early childhoodExtreme and constant hunger (hyperphagia)Rapid weight gainDifficulty feeling full after eatingMild intellectual disability or learning difficultiesBehavioral problems such as stubbornness or emotional outburstsFeatures resembling Prader-Willi syndromeIncreased risk of type 2 diabetesHigh blood pressureSleep apneaJoint pain or mobility problems due to excess weightLow muscle tone in infancy

Clinical phenotype terms (17)— hover any for plain English
Postural hypotension with compensatory tachycardiaHP:0005307Decreased circulating vitamin B1 concentrationHP:0100503Glucose intoleranceHP:0001952Increased resting energy expenditureHP:0012339
Inheritance

Autosomal dominant

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

Age of Onset

Childhood

Begins in childhood, roughly ages 1 to 12

Orphanet ↗NORD ↗

Treatments

No FDA-approved treatments are currently listed for Obesity due to SIM1 deficiency.

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No actively recruiting trials found for Obesity due to SIM1 deficiency at this time.

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Search ClinicalTrials.gov ↗Join the Obesity due to SIM1 deficiency community →

No specialists are currently listed for Obesity due to SIM1 deficiency.

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 Obesity due to SIM1 deficiency.

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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.

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Family & Caregiver Grants

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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.Is my child's obesity caused by a change in the SIM1 gene, and how was this confirmed?,Are there any medications, such as setmelanotide, that might help control my child's hunger?,What kind of diet and exercise plan do you recommend for my child?,Should we screen for complications like diabetes, high blood pressure, or sleep apnea, and how often?,Are there clinical trials or new treatments being studied for SIM1-related obesity?,Should other family members be tested for changes in the SIM1 gene?,Can you refer us to a behavioral psychologist experienced with genetic obesity?

Common questions about Obesity due to SIM1 deficiency

What is Obesity due to SIM1 deficiency?

Obesity due to SIM1 deficiency is a rare genetic condition caused by changes (mutations) in the SIM1 gene. SIM1 stands for Single-Minded Homolog 1, and this gene plays an important role in the development and function of a part of the brain called the hypothalamus. The hypothalamus is the brain's control center for hunger, fullness, and energy balance. When the SIM1 gene does not work properly, the brain has trouble regulating appetite, which leads to severe early-onset obesity. Children with this condition typically develop an intense, hard-to-control hunger (called hyperphagia) starting in

How is Obesity due to SIM1 deficiency inherited?

Obesity due to SIM1 deficiency follows a autosomal dominant inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.

At what age does Obesity due to SIM1 deficiency typically begin?

Typical onset of Obesity due to SIM1 deficiency is childhood. Age of onset can vary across affected individuals.