X-linked endothelial corneal dystrophy

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ORPHA:293621OMIM:300779H18.5
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Overview

X-linked endothelial corneal dystrophy (XECD) is a very rare inherited eye condition that affects the cornea, which is the clear front window of the eye. This disease primarily impacts the endothelial cells, which form the innermost layer of the cornea. These cells are responsible for keeping the cornea clear by pumping excess fluid out of it. When these cells do not work properly, the cornea becomes swollen and cloudy, leading to vision problems. Because this condition is X-linked, it mainly affects males, though females who carry the gene change may show milder symptoms. Males typically present with significant corneal clouding (also called corneal edema) that can be noticed at birth or in early infancy. The clouding can affect one or both eyes and may be accompanied by a moon crater-like appearance of the endothelial cells when viewed under a special microscope (specular microscopy). Some affected individuals may also develop nystagmus (involuntary eye movements) due to poor vision early in life. There is currently no cure for XECD. Treatment focuses on managing symptoms and preserving vision. Corneal transplant surgery (keratoplasty) may be considered in severe cases to replace the damaged cornea with a healthy donor cornea. Hypertonic saline eye drops may help reduce corneal swelling temporarily. Early intervention is important to prevent amblyopia (lazy eye) in young children.

Also known as:

Key symptoms:

Cloudy or hazy corneasBlurred or reduced visionCorneal swelling (edema)Involuntary eye movements (nystagmus)Ground-glass appearance of the corneaVision problems from birth or early infancySensitivity to lightTearing of the eyesPossible lazy eye (amblyopia) if untreated

Clinical phenotype terms (7)— hover any for plain English
Band keratopathyHP:0000585Abnormal corneal endothelium morphologyHP:0011488Nuclear cataractHP:0100018
Inheritance

X-linked dominant

Carried on the X chromosome; a single copy can cause the condition

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 X-linked endothelial corneal dystrophy.

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No actively recruiting trials found for X-linked endothelial corneal dystrophy at this time.

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No specialists are currently listed for X-linked endothelial corneal dystrophy.

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 X-linked endothelial corneal dystrophy.

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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.How severe is the corneal clouding, and how much is it affecting my child's vision?,Is corneal transplant surgery recommended now, or should we wait and monitor?,What type of corneal transplant would be best, and what are the risks?,Are there eye drops or other treatments that can help in the meantime?,Should we be concerned about amblyopia, and what steps can we take to prevent it?,Should other family members be examined for signs of this condition?,Is genetic testing available to confirm the diagnosis and identify carriers in the family?

Common questions about X-linked endothelial corneal dystrophy

What is X-linked endothelial corneal dystrophy?

X-linked endothelial corneal dystrophy (XECD) is a very rare inherited eye condition that affects the cornea, which is the clear front window of the eye. This disease primarily impacts the endothelial cells, which form the innermost layer of the cornea. These cells are responsible for keeping the cornea clear by pumping excess fluid out of it. When these cells do not work properly, the cornea becomes swollen and cloudy, leading to vision problems. Because this condition is X-linked, it mainly affects males, though females who carry the gene change may show milder symptoms. Males typically pre

How is X-linked endothelial corneal dystrophy inherited?

X-linked endothelial corneal dystrophy follows a x-linked dominant inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.

At what age does X-linked endothelial corneal dystrophy typically begin?

Typical onset of X-linked endothelial corneal dystrophy is neonatal. Age of onset can vary across affected individuals.