Short stature-intellectual disability-eye anomalies-cleft lip/palate syndrome

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ORPHA:2649OMIM:243310
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1FDA treatments8Treatment centers1Financial resources

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Overview

Short stature-intellectual disability-eye anomalies-cleft lip/palate syndrome is an extremely rare genetic condition that affects multiple parts of the body. As the name describes, people with this syndrome typically have shorter-than-expected height, some degree of intellectual disability or learning difficulties, problems with the eyes, and a cleft lip and/or cleft palate (an opening in the upper lip or roof of the mouth present at birth). The eye problems can include small eyes (microphthalmia), clouding of the lens (cataracts), or other structural abnormalities of the eye that may affect vision. This condition was first described in a small number of families, and very few cases have been reported in the medical literature. Because of its rarity, the full range of symptoms and their severity can vary from person to person. Some individuals may have additional features such as distinctive facial characteristics or other developmental differences. There is currently no cure for this syndrome. Treatment focuses on managing each symptom individually. This may include growth hormone evaluation for short stature, surgical repair of cleft lip or palate, eye surgeries or corrective lenses for vision problems, and special education services or therapies for intellectual disability. A team of specialists working together is important to provide the best possible care and quality of life for affected individuals.

Also known as:

Key symptoms:

Short stature or growth delayIntellectual disability or learning difficultiesEye abnormalities such as small eyes or cataractsCleft lip (opening in the upper lip)Cleft palate (opening in the roof of the mouth)Distinctive facial featuresVision problems or vision lossDelayed developmental milestonesSpeech and language delaysFeeding difficulties in infancy

Inheritance

Autosomal recessive

Passed on when both parents carry the same gene change; often skips generations

Age of Onset

Neonatal

Begins at or shortly after birth (first 4 weeks)

Orphanet ↗OMIM ↗NORD ↗

Treatments

1 available

Sogroya

somapacitan-beco· Novo Nordisk Pharmaceuticals

Short stature born small for gestational age (SGA) and with no catch-up growth by 2 years of age

No actively recruiting trials found for Short stature-intellectual disability-eye anomalies-cleft lip/palate syndrome at this time.

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

Search ClinicalTrials.gov ↗Join the Short stature-intellectual disability-eye anomalies-cleft lip/palate syndrome community →

No specialists are currently listed for Short stature-intellectual disability-eye anomalies-cleft lip/palate 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

Financial Resources

1 resources
Sogroya(somapacitan-beco)Novo Nordisk Pharmaceuticals

Travel Grants

No travel grants are currently matched to Short stature-intellectual disability-eye anomalies-cleft lip/palate syndrome.

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Community

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Latest news about Short stature-intellectual disability-eye anomalies-cleft lip/palate syndrome

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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 specific eye problems does my child have, and how will they be treated?,When is the best time to schedule cleft lip and palate repair surgery?,Should my child be evaluated for growth hormone deficiency?,What early intervention therapies do you recommend, and how soon should we start?,Is genetic testing available to identify the exact cause of this condition?,How often should my child have eye exams and hearing tests?,What educational supports should we put in place as my child grows?

Common questions about Short stature-intellectual disability-eye anomalies-cleft lip/palate syndrome

What is Short stature-intellectual disability-eye anomalies-cleft lip/palate syndrome?

Short stature-intellectual disability-eye anomalies-cleft lip/palate syndrome is an extremely rare genetic condition that affects multiple parts of the body. As the name describes, people with this syndrome typically have shorter-than-expected height, some degree of intellectual disability or learning difficulties, problems with the eyes, and a cleft lip and/or cleft palate (an opening in the upper lip or roof of the mouth present at birth). The eye problems can include small eyes (microphthalmia), clouding of the lens (cataracts), or other structural abnormalities of the eye that may affect v

How is Short stature-intellectual disability-eye anomalies-cleft lip/palate syndrome inherited?

Short stature-intellectual disability-eye anomalies-cleft lip/palate syndrome follows a autosomal recessive inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.

At what age does Short stature-intellectual disability-eye anomalies-cleft lip/palate syndrome typically begin?

Typical onset of Short stature-intellectual disability-eye anomalies-cleft lip/palate syndrome is neonatal. Age of onset can vary across affected individuals.