Oculotrichodysplasia

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ORPHA:2718OMIM:257960Q82.4
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Overview

Oculotrichodysplasia is a very rare inherited condition that affects the eyes and hair. The name comes from the Latin and Greek words for eye (oculo), hair (tricho), and abnormal development (dysplasia). It is sometimes referred to in medical literature simply by its Orphanet code or as part of a broader group of ectodermal dysplasias — conditions where tissues that develop from the outer layer of the embryo (the ectoderm) do not form normally. The main features of this condition involve problems with the eyes, such as small eyes (microphthalmia), clouding of the lens (cataracts), or other structural eye abnormalities that can affect vision. Hair abnormalities are also a core feature — the hair may be sparse, fragile, unusually textured, or slow to grow. Some individuals may also have changes in the nails or skin, since these tissues share a similar developmental origin. Because this condition is extremely rare, the full range of symptoms and how they vary between individuals is not yet completely understood. Treatment focuses on managing specific symptoms, such as correcting vision problems with glasses, contact lenses, or surgery, and supporting hair and skin health. There is currently no cure. A clinical geneticist and a team of specialists are key to guiding care.

Also known as:

Key symptoms:

Abnormally small eyes (microphthalmia)Clouding of the eye lens (cataracts)Reduced or poor visionSparse or thin hairFragile or brittle hair that breaks easilySlow hair growthUnusual hair texture or structurePossible nail abnormalitiesPossible skin changes or dryness

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 Oculotrichodysplasia.

View clinical trials →

No actively recruiting trials found for Oculotrichodysplasia at this time.

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

Search ClinicalTrials.gov ↗Join the Oculotrichodysplasia community →

No specialists are currently listed for Oculotrichodysplasia.

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 Oculotrichodysplasia.

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Community

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Latest news about Oculotrichodysplasia

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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 confirm the diagnosis and find the gene responsible?,How severe is my child's vision problem, and what treatments are available?,Will the eye problems get worse over time, and how often should we have eye check-ups?,Are other family members at risk, and should they be tested?,What support services or vision rehabilitation programs are available for my child?,Are there any clinical trials or research studies we could participate in?,What should we tell our child's school about their needs?

Common questions about Oculotrichodysplasia

What is Oculotrichodysplasia?

Oculotrichodysplasia is a very rare inherited condition that affects the eyes and hair. The name comes from the Latin and Greek words for eye (oculo), hair (tricho), and abnormal development (dysplasia). It is sometimes referred to in medical literature simply by its Orphanet code or as part of a broader group of ectodermal dysplasias — conditions where tissues that develop from the outer layer of the embryo (the ectoderm) do not form normally. The main features of this condition involve problems with the eyes, such as small eyes (microphthalmia), clouding of the lens (cataracts), or other st

How is Oculotrichodysplasia inherited?

Oculotrichodysplasia follows a autosomal recessive inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.

At what age does Oculotrichodysplasia typically begin?

Typical onset of Oculotrichodysplasia is neonatal. Age of onset can vary across affected individuals.