Pseudoxanthoma elasticum-like skin manifestations with retinitis pigmentosa

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Overview

Pseudoxanthoma elasticum-like skin manifestations with retinitis pigmentosa is an extremely rare genetic condition that combines two main problems: skin changes that look like a disease called pseudoxanthoma elasticum (PXE) and a progressive eye condition called retinitis pigmentosa (RP). The skin changes include yellowish, small bumps and loose, sagging skin, especially in areas like the neck, armpits, and groin. These changes happen because the elastic fibers in the skin become damaged and calcified, making the skin look aged or wrinkled. At the same time, retinitis pigmentosa causes a gradual loss of vision. It starts with difficulty seeing in dim light (night blindness) and a narrowing of the visual field (tunnel vision), and can eventually lead to significant vision loss over time. This condition is distinct from classic pseudoxanthoma elasticum, which is caused by mutations in the ABCC6 gene. Instead, this combined syndrome has been linked to other genetic causes. Because it is so rare, treatment is mainly supportive and focused on managing symptoms. There is no cure at this time. Eye care to slow vision loss and skin care to manage the cosmetic effects are the main approaches. Regular monitoring by both eye specialists and dermatologists is important to track how the disease progresses and to address complications as they arise.

Also known as:

Key symptoms:

Yellowish bumps on the skin, especially on the neck, armpits, and groinLoose, sagging, or wrinkled skinNight blindness or difficulty seeing in low lightTunnel vision or narrowing of the visual fieldProgressive vision lossSkin that looks prematurely agedCalcification of elastic fibers in the skinPossible angioid streaks in the back of the eyeSkin laxity in flexural areas (skin folds)Gradual loss of peripheral vision

Clinical phenotype terms (11)— hover any for plain English
Increased number of skin foldsHP:0007522Attenuation of retinal blood vesselsHP:0007843Absent retinal pigment epitheliumHP:0007980Abnormal fundus morphologyHP:0001098
Inheritance

Autosomal recessive

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

Age of Onset

Variable

Can begin at different ages, from infancy through adulthood

Orphanet ↗NORD ↗

Treatments

No FDA-approved treatments are currently listed for Pseudoxanthoma elasticum-like skin manifestations with retinitis pigmentosa.

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No actively recruiting trials found for Pseudoxanthoma elasticum-like skin manifestations with retinitis pigmentosa at this time.

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No specialists are currently listed for Pseudoxanthoma elasticum-like skin manifestations with retinitis pigmentosa.

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 Pseudoxanthoma elasticum-like skin manifestations with retinitis pigmentosa.

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Community

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Caregiver Resources

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Questions for your doctor

Bring these to your next appointment

  • Q1.What is the current status of my vision, and how quickly might it change?,Are there any treatments or clinical trials that could slow my vision loss?,How often should I have eye exams and skin evaluations?,Should my family members be tested for this condition?,What low-vision aids or assistive devices would you recommend for me?,Are there any lifestyle changes that could help protect my skin or eyes?,Can you refer me to a genetic counselor to discuss family planning?

Common questions about Pseudoxanthoma elasticum-like skin manifestations with retinitis pigmentosa

What is Pseudoxanthoma elasticum-like skin manifestations with retinitis pigmentosa?

Pseudoxanthoma elasticum-like skin manifestations with retinitis pigmentosa is an extremely rare genetic condition that combines two main problems: skin changes that look like a disease called pseudoxanthoma elasticum (PXE) and a progressive eye condition called retinitis pigmentosa (RP). The skin changes include yellowish, small bumps and loose, sagging skin, especially in areas like the neck, armpits, and groin. These changes happen because the elastic fibers in the skin become damaged and calcified, making the skin look aged or wrinkled. At the same time, retinitis pigmentosa causes a gradu

How is Pseudoxanthoma elasticum-like skin manifestations with retinitis pigmentosa inherited?

Pseudoxanthoma elasticum-like skin manifestations with retinitis pigmentosa follows a autosomal recessive inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.