Severe growth deficiency-strabismus-extensive dermal melanocytosis-intellectual disability syndrome

Last reviewed

🖨 Print for my doctorAdvocacy Hub →
ORPHA:488627OMIM:617051Q87.8
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

Severe growth deficiency-strabismus-extensive dermal melanocytosis-intellectual disability syndrome is a very rare genetic condition that affects multiple parts of the body from birth. It is sometimes referred to by its Orphanet code ORPHA:488627. The condition is defined by four main features: significantly shorter height and slower growth than expected for age, crossed or misaligned eyes (called strabismus), large areas of bluish-gray skin discoloration caused by pigment cells deep in the skin (called extensive dermal melanocytosis, similar to but more widespread than common 'Mongolian spots'), and intellectual disability that affects learning, communication, and daily functioning. This syndrome appears to be caused by changes in a person's genetic material, though research is still ongoing to fully understand the underlying cause. Because it is so rare, most of what is known comes from a very small number of reported cases. There is currently no cure for this condition. Treatment focuses on managing each symptom individually — for example, glasses or surgery for eye misalignment, educational support and therapy for intellectual disability, and regular monitoring of growth. A team of different specialists is usually needed to provide the best care.

Key symptoms:

Significantly shorter height and slower growth than other children the same ageCrossed or misaligned eyes (strabismus)Large areas of bluish-gray skin discoloration, especially on the back and buttocks, caused by pigment cells deep in the skinIntellectual disability affecting learning, memory, and communicationDelayed developmental milestones such as sitting, walking, and talkingPossible facial features that look different from other family members

Clinical phenotype terms (40)— hover any for plain English
Multifocal cerebral white matter abnormalitiesHP:0007052Bilateral tonic-clonic seizure with focal onsetHP:0007334Decreased glomerular filtration rateHP:0012213
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

No FDA-approved treatments are currently listed for Severe growth deficiency-strabismus-extensive dermal melanocytosis-intellectual disability syndrome.

View clinical trials →

No actively recruiting trials found for Severe growth deficiency-strabismus-extensive dermal melanocytosis-intellectual disability syndrome at this time.

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

Search ClinicalTrials.gov ↗Join the Severe growth deficiency-strabismus-extensive dermal melanocytosis-intellectual disability syndrome community →

No specialists are currently listed for Severe growth deficiency-strabismus-extensive dermal melanocytosis-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 Severe growth deficiency-strabismus-extensive dermal melanocytosis-intellectual disability syndrome.

Search all travel grants →NORD Financial Assistance ↗

Community

Open Severe growth deficiency-strabismus-extensive dermal melanocytosis-intellectual disability syndromeForum →

No community posts yet. Be the first to share your experience with Severe growth deficiency-strabismus-extensive dermal melanocytosis-intellectual disability syndrome.

Start the conversation →

Latest news about Severe growth deficiency-strabismus-extensive dermal melanocytosis-intellectual disability syndrome

No recent news articles for Severe growth deficiency-strabismus-extensive dermal melanocytosis-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 genetic tests should we do to try to find the cause of my child's condition?,Which specialists does my child need to see, and how often?,What early intervention or therapy programs would help my child the most right now?,Is my child's growth problem related to a hormone deficiency that could be treated?,What should we watch for that would mean my child needs urgent medical attention?,Are there any research studies or registries for this condition that we could join?,What support services are available to help our family manage day-to-day care?

Common questions about Severe growth deficiency-strabismus-extensive dermal melanocytosis-intellectual disability syndrome

What is Severe growth deficiency-strabismus-extensive dermal melanocytosis-intellectual disability syndrome?

Severe growth deficiency-strabismus-extensive dermal melanocytosis-intellectual disability syndrome is a very rare genetic condition that affects multiple parts of the body from birth. It is sometimes referred to by its Orphanet code ORPHA:488627. The condition is defined by four main features: significantly shorter height and slower growth than expected for age, crossed or misaligned eyes (called strabismus), large areas of bluish-gray skin discoloration caused by pigment cells deep in the skin (called extensive dermal melanocytosis, similar to but more widespread than common 'Mongolian spots

How is Severe growth deficiency-strabismus-extensive dermal melanocytosis-intellectual disability syndrome inherited?

Severe growth deficiency-strabismus-extensive dermal melanocytosis-intellectual disability syndrome follows a autosomal recessive inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.

At what age does Severe growth deficiency-strabismus-extensive dermal melanocytosis-intellectual disability syndrome typically begin?

Typical onset of Severe growth deficiency-strabismus-extensive dermal melanocytosis-intellectual disability syndrome is neonatal. Age of onset can vary across affected individuals.