Familial drusen

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ORPHA:75376OMIM:126600H35.5
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Overview

Familial drusen, also known as Doyne honeycomb retinal dystrophy, Malattia Leventinese, or autosomal dominant drusen, is a rare inherited eye condition that affects the retina — the light-sensitive layer at the back of your eye. The disease causes small yellowish-white deposits called drusen to build up under the retina, particularly in the central area called the macula. These deposits interfere with how the retina works and can damage the cells responsible for sharp, detailed vision. Most people with familial drusen begin to notice symptoms in their 20s to 40s, though the timing can vary. Early on, you might notice that straight lines look wavy, or that your central vision seems blurry or distorted. Over time, the drusen deposits can grow and spread, and some people develop more serious complications like abnormal blood vessel growth under the retina (called choroidal neovascularization), which can cause rapid vision loss. Side (peripheral) vision is usually preserved. There is currently no cure for familial drusen. Treatment focuses on monitoring the eyes regularly and, if complications like abnormal blood vessels develop, using anti-VEGF injections (such as ranibizumab or bevacizumab) to slow further damage. Low vision aids and lifestyle adjustments can help people manage daily tasks. Research into gene therapy and other targeted treatments is ongoing, giving hope for better options in the future.

Also known as:

Key symptoms:

Blurry or distorted central visionStraight lines appearing wavy or bent (metamorphopsia)Difficulty reading fine printReduced ability to see fine detailYellowish-white spots (drusen) visible on eye examinationGradual worsening of central vision over timeDifficulty recognizing facesSensitivity to bright lightDifficulty adapting from bright to dim lightIn advanced cases, a blind spot in the center of vision

Clinical phenotype terms (20)— hover any for plain English
Macular drusenHP:0030499Yellow/white macular lesionHP:0030500Macular dystrophyHP:0007754Reticular pigmentary degenerationHP:0007937Macular hyperpigmentationHP:0011509MetamorphopsiaHP:0012508Perifoveal ring of hyperautofluorescenceHP:0030629Hyperautofluorescent macular lesionHP:0030631Hypoautofluorescent macular lesionHP:0030632Macular atrophyHP:0007401Granular macular appearanceHP:0007793Peripapillary chorioretinal atrophyHP:0007950Choroidal neovascularizationHP:0011506Serous retinal detachmentHP:0012231Macular hemorrhageHP:0025574Paracentral scotomaHP:0030528Subretinal fluidHP:0031526
Inheritance

Autosomal dominant

Passed on from just one parent; each child has about a 50% chance of inheriting it

Age of Onset

Adult

Begins in adulthood (age 18 or older)

Orphanet ↗OMIM ↗NORD ↗

Treatments

No FDA-approved treatments are currently listed for Familial drusen.

View clinical trials →

No actively recruiting trials found for Familial drusen at this time.

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

Search ClinicalTrials.gov ↗Join the Familial drusen community →

No specialists are currently listed for Familial drusen.

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 Familial drusen.

Search all travel grants →NORD Financial Assistance ↗

Community

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Latest news about Familial drusen

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Caregiver Resources

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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 advanced is my condition right now, and how quickly do you expect it to progress?,Should I have genetic testing, and should my family members be tested too?,How often should I come in for monitoring, and what signs should make me call sooner?,Should I be using an Amsler grid at home, and how do I use it correctly?,Are there any vitamin supplements or lifestyle changes that might help slow the disease?,What are the warning signs that I need anti-VEGF injections, and how quickly would I need to start them?,Are there any clinical trials or research studies I could join?

Common questions about Familial drusen

What is Familial drusen?

Familial drusen, also known as Doyne honeycomb retinal dystrophy, Malattia Leventinese, or autosomal dominant drusen, is a rare inherited eye condition that affects the retina — the light-sensitive layer at the back of your eye. The disease causes small yellowish-white deposits called drusen to build up under the retina, particularly in the central area called the macula. These deposits interfere with how the retina works and can damage the cells responsible for sharp, detailed vision. Most people with familial drusen begin to notice symptoms in their 20s to 40s, though the timing can vary. E

How is Familial drusen inherited?

Familial drusen follows a autosomal dominant inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.

At what age does Familial drusen typically begin?

Typical onset of Familial drusen is adult. Age of onset can vary across affected individuals.