Ectodermal dysplasia-blindness syndrome

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ORPHA:1806OMIM:268320Q87.8
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Overview

Ectodermal dysplasia-blindness syndrome is an extremely rare genetic condition that combines features of ectodermal dysplasia with severe vision problems leading to blindness. Ectodermal dysplasia refers to a group of disorders that affect the development of structures that come from the outer layer of the embryo (the ectoderm), including the skin, hair, nails, teeth, and sweat glands. In this particular syndrome, affected individuals typically have sparse or abnormal hair, problems with their teeth (such as missing or malformed teeth), reduced ability to sweat, and skin abnormalities. The hallmark feature that distinguishes this syndrome from other forms of ectodermal dysplasia is progressive vision loss that can lead to blindness, often due to abnormalities in the structures of the eye. Because this condition is so rare, the treatment landscape is limited and primarily focused on managing symptoms rather than curing the underlying cause. Eye care specialists work to preserve vision as long as possible, while dental specialists address tooth abnormalities. Skin care and temperature regulation are important for individuals who have reduced sweating. Genetic counseling is recommended for affected families to understand the inheritance pattern and risks for future children. Research into this condition is very limited due to its extreme rarity, and much of what is known comes from a small number of reported cases in the medical literature.

Key symptoms:

Severe vision loss or blindnessSparse or thin hairMissing or abnormally shaped teethReduced ability to sweatDry skinAbnormal or thin nailsEye abnormalities present from birthSensitivity to heat due to poor sweatingThin or fragile skinPossible intellectual disability in some cases

Clinical phenotype terms (25)— hover any for plain English
SclerocorneaHP:0000647
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 Ectodermal dysplasia-blindness syndrome.

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No actively recruiting trials found for Ectodermal dysplasia-blindness syndrome at this time.

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No specialists are currently listed for Ectodermal dysplasia-blindness 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 Ectodermal dysplasia-blindness 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 is the expected course of my child's vision loss, and are there any treatments that could slow it down?,Should we pursue genetic testing, and what type of testing do you recommend?,How can we best manage the risk of overheating due to reduced sweating?,What dental treatments are appropriate and when should they begin?,Are there any clinical trials or research studies we could participate in?,What vision support services and assistive technologies are available?,What is the chance that future children in our family could be affected?

Common questions about Ectodermal dysplasia-blindness syndrome

What is Ectodermal dysplasia-blindness syndrome?

Ectodermal dysplasia-blindness syndrome is an extremely rare genetic condition that combines features of ectodermal dysplasia with severe vision problems leading to blindness. Ectodermal dysplasia refers to a group of disorders that affect the development of structures that come from the outer layer of the embryo (the ectoderm), including the skin, hair, nails, teeth, and sweat glands. In this particular syndrome, affected individuals typically have sparse or abnormal hair, problems with their teeth (such as missing or malformed teeth), reduced ability to sweat, and skin abnormalities. The hal

How is Ectodermal dysplasia-blindness syndrome inherited?

Ectodermal dysplasia-blindness syndrome follows a autosomal recessive inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.

At what age does Ectodermal dysplasia-blindness syndrome typically begin?

Typical onset of Ectodermal dysplasia-blindness syndrome is neonatal. Age of onset can vary across affected individuals.