Olivopontocerebellar atrophy-deafness syndrome

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Overview

Olivopontocerebellar atrophy-deafness syndrome (also known as OPCA with deafness or Olivopontocerebellar atrophy with sensorineural deafness) is an extremely rare inherited neurological condition that affects the brain and hearing. In this disease, certain parts of the brain — specifically the olivary nuclei, the pons, and the cerebellum — gradually shrink and lose function. These brain regions are responsible for coordinating movement, balance, and other important functions. As a result, affected individuals develop progressive problems with coordination (ataxia), balance difficulties, and hearing loss due to damage to the nerves that carry sound signals from the ear to the brain (sensorineural deafness). Symptoms typically include difficulty walking and maintaining balance, slurred speech, involuntary eye movements, and progressive hearing loss. The condition tends to worsen over time as the brain structures continue to deteriorate. Some individuals may also experience difficulty swallowing and changes in muscle tone. There is currently no cure for this condition. Treatment focuses on managing symptoms and maintaining quality of life. This may include physical therapy to help with balance and coordination, hearing aids or cochlear implants for hearing loss, speech therapy, and occupational therapy. A team of specialists is usually needed to provide comprehensive care.

Also known as:

Key symptoms:

Progressive loss of coordination (ataxia)Difficulty walking and balance problemsHearing loss that worsens over timeSlurred or unclear speechInvoluntary eye movements (nystagmus)Difficulty swallowingMuscle stiffness or spasticityTremorsUnsteady gaitLoss of fine motor skillsDizzinessCognitive changes in some cases

Clinical phenotype terms (14)— hover any for plain English
Olivopontocerebellar atrophyHP:0002542
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 Olivopontocerebellar atrophy-deafness syndrome.

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No actively recruiting trials found for Olivopontocerebellar atrophy-deafness syndrome at this time.

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Search ClinicalTrials.gov ↗Join the Olivopontocerebellar atrophy-deafness syndrome community →

No specialists are currently listed for Olivopontocerebellar atrophy-deafness 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 Olivopontocerebellar atrophy-deafness syndrome.

Search all travel grants →NORD Financial Assistance ↗

Community

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Latest news about Olivopontocerebellar atrophy-deafness syndrome

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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 quickly is this condition likely to progress in my case or my child's case?,What therapies are most important to start right away?,Should we pursue genetic testing, and what can it tell us about our family's risk?,What hearing interventions would be most helpful at this stage?,Are there any clinical trials or research studies we could participate in?,What signs should prompt us to seek urgent medical attention?,How can we best plan for future mobility and communication needs?

Common questions about Olivopontocerebellar atrophy-deafness syndrome

What is Olivopontocerebellar atrophy-deafness syndrome?

Olivopontocerebellar atrophy-deafness syndrome (also known as OPCA with deafness or Olivopontocerebellar atrophy with sensorineural deafness) is an extremely rare inherited neurological condition that affects the brain and hearing. In this disease, certain parts of the brain — specifically the olivary nuclei, the pons, and the cerebellum — gradually shrink and lose function. These brain regions are responsible for coordinating movement, balance, and other important functions. As a result, affected individuals develop progressive problems with coordination (ataxia), balance difficulties, and he

How is Olivopontocerebellar atrophy-deafness syndrome inherited?

Olivopontocerebellar atrophy-deafness syndrome follows a autosomal recessive inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.