Cochleosaccular degeneration-cataract syndrome

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ORPHA:3233OMIM:120040H83.8
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Overview

Cochleosaccular degeneration-cataract syndrome, also known as Norrie-like disease or sometimes referred to as Scheie-like cochleosaccular degeneration with cataracts, is an extremely rare genetic condition that affects both hearing and vision. The syndrome involves two main problems: degeneration of parts of the inner ear (specifically the cochlea and saccule, which are responsible for hearing and balance) and the development of cataracts (clouding of the lens in the eye). The hearing loss in this condition is sensorineural, meaning it results from damage to the inner ear structures rather than the outer or middle ear. It typically affects both ears and can range from moderate to profound. The cataracts may develop early in life and can significantly impair vision if not treated. Some affected individuals may also experience balance difficulties due to the involvement of the saccule, which plays a role in detecting gravity and movement. Because this syndrome is so rare, treatment is mainly supportive. Hearing aids or cochlear implants may help with hearing loss, and cataract surgery can restore vision. There is currently no cure or treatment that can stop or reverse the underlying degeneration of the inner ear structures. Early diagnosis and intervention are important to optimize hearing and visual outcomes, especially in children during critical periods of speech and language development.

Key symptoms:

Hearing loss in both earsClouding of the eye lens (cataracts)Balance problems or dizzinessDifficulty understanding speechProgressive worsening of hearingBlurred or hazy visionSensitivity to light or glareDelayed speech development in childrenDifficulty with coordination

Clinical phenotype terms (5)— hover any for plain English
Progressive sensorineural hearing impairmentHP:0000408Cochlear degenerationHP:0005102
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 ↗OMIM ↗NORD ↗

Treatments

No FDA-approved treatments are currently listed for Cochleosaccular degeneration-cataract syndrome.

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No actively recruiting trials found for Cochleosaccular degeneration-cataract syndrome at this time.

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No specialists are currently listed for Cochleosaccular degeneration-cataract syndrome.

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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 Cochleosaccular degeneration-cataract syndrome.

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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.How severe is the hearing loss, and is it expected to get worse over time?,When should cataract surgery be considered, and what are the risks and benefits?,Would hearing aids or cochlear implants be the best option for managing hearing loss?,Are there genetic tests available that could confirm the diagnosis or help with family planning?,What therapies or support services should we start right away?,How often should hearing and vision be monitored?,Are there any clinical trials or new treatments being studied for this condition?

Common questions about Cochleosaccular degeneration-cataract syndrome

What is Cochleosaccular degeneration-cataract syndrome?

Cochleosaccular degeneration-cataract syndrome, also known as Norrie-like disease or sometimes referred to as Scheie-like cochleosaccular degeneration with cataracts, is an extremely rare genetic condition that affects both hearing and vision. The syndrome involves two main problems: degeneration of parts of the inner ear (specifically the cochlea and saccule, which are responsible for hearing and balance) and the development of cataracts (clouding of the lens in the eye). The hearing loss in this condition is sensorineural, meaning it results from damage to the inner ear structures rather th

How is Cochleosaccular degeneration-cataract syndrome inherited?

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