Retinal degeneration-nanophthalmos-glaucoma syndrome

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ORPHA:1574OMIM:267760H35.5
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1FDA treatments8Treatment centers

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Overview

Retinal degeneration-nanophthalmos-glaucoma syndrome is an extremely rare inherited eye condition that combines three major eye problems. First, the eyes are unusually small (a condition called nanophthalmos), which means the eyeball is shorter than normal from front to back. Second, the retina — the light-sensitive layer at the back of the eye — gradually breaks down over time (retinal degeneration), leading to progressive vision loss. Third, the condition causes glaucoma, which is a buildup of pressure inside the eye that can damage the optic nerve and further threaten vision. Because the eyes are abnormally small, the internal structures are crowded together. This crowding makes it harder for fluid to drain properly from the eye, which contributes to the development of glaucoma. The retinal degeneration typically affects both eyes and can lead to night blindness, tunnel vision, and eventually significant vision impairment. Patients may also be very farsighted (hyperopic) due to the small size of their eyes. There is currently no cure for this syndrome. Treatment focuses on managing each component: lowering eye pressure with medications or surgery to protect against glaucoma damage, and supportive measures for vision loss caused by retinal degeneration. Regular monitoring by eye specialists is essential to preserve as much vision as possible for as long as possible. Low-vision aids and rehabilitation services can help patients maintain independence.

Also known as:

Key symptoms:

Unusually small eyesSevere farsightednessProgressive vision lossNight blindnessTunnel vision or loss of side visionIncreased eye pressure (glaucoma)Eye pain or headaches from high eye pressureDifficulty seeing in dim lightingBlurred or hazy visionGradual loss of central vision in later stages

Clinical phenotype terms (7)— hover any for plain English
Inheritance

Autosomal recessive

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

Age of Onset

Childhood

Begins in childhood, roughly ages 1 to 12

Orphanet ↗OMIM ↗NORD ↗

FDA & Trial Timeline

1 event
Feb 2009

MembraneBlue: FDA approved

For use as an aid in ophthalmic surgery by staining the epiretinal membranes during ophthalmic surgical vitrectomy procedures, facilitating removal of the tissue

FDAcompleted

Data sourced from FDA regulatory filings and ClinicalTrials.gov. Updated periodically.

Treatments

1 available

MembraneBlue

Trypan blue· Dutch Ophthalmic Research Center International BVOrphan Drug

For use as an aid in ophthalmic surgery by staining the epiretinal membranes during ophthalmic surgical vitrectomy procedures, facilitating removal of the tissue

No actively recruiting trials found for Retinal degeneration-nanophthalmos-glaucoma syndrome at this time.

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

Search ClinicalTrials.gov ↗Join the Retinal degeneration-nanophthalmos-glaucoma syndrome community →

No specialists are currently listed for Retinal degeneration-nanophthalmos-glaucoma 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 Retinal degeneration-nanophthalmos-glaucoma syndrome.

Search all travel grants →NORD Financial Assistance ↗

Community

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Latest news about Retinal degeneration-nanophthalmos-glaucoma syndrome

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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.How advanced is the retinal degeneration in my eyes right now, and how quickly might it progress?,What is my current eye pressure, and is my glaucoma well controlled?,What are the safest treatment options for glaucoma given that my eyes are unusually small?,Should I or my family members undergo genetic testing, and what would the results mean for us?,Are there any clinical trials or emerging therapies, such as gene therapy, that I might be eligible for?,What low-vision aids or rehabilitation services would you recommend for my current level of vision?,How often should I have my eyes checked, and what warning signs should prompt an urgent visit?

Common questions about Retinal degeneration-nanophthalmos-glaucoma syndrome

What is Retinal degeneration-nanophthalmos-glaucoma syndrome?

Retinal degeneration-nanophthalmos-glaucoma syndrome is an extremely rare inherited eye condition that combines three major eye problems. First, the eyes are unusually small (a condition called nanophthalmos), which means the eyeball is shorter than normal from front to back. Second, the retina — the light-sensitive layer at the back of the eye — gradually breaks down over time (retinal degeneration), leading to progressive vision loss. Third, the condition causes glaucoma, which is a buildup of pressure inside the eye that can damage the optic nerve and further threaten vision. Because the e

How is Retinal degeneration-nanophthalmos-glaucoma syndrome inherited?

Retinal degeneration-nanophthalmos-glaucoma syndrome follows a autosomal recessive inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.

At what age does Retinal degeneration-nanophthalmos-glaucoma syndrome typically begin?

Typical onset of Retinal degeneration-nanophthalmos-glaucoma syndrome is childhood. Age of onset can vary across affected individuals.

What treatment and support options exist for Retinal degeneration-nanophthalmos-glaucoma syndrome?

1 patient support program are currently tracked on UniteRare for Retinal degeneration-nanophthalmos-glaucoma syndrome. See the treatments and support programs sections for copay assistance, eligibility, and contact details.