Autosomal recessive cerebellar ataxia-pyramidal signs-nystagmus-oculomotor apraxia syndrome

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ORPHA:363429OMIM:614831G11.1
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Overview

Autosomal recessive cerebellar ataxia with pyramidal signs, nystagmus, and oculomotor apraxia syndrome is a very rare inherited neurological condition that affects the brain's ability to coordinate movement. The disease primarily impacts the cerebellum, which is the part of the brain responsible for balance and smooth, coordinated movements. People with this condition develop cerebellar ataxia, meaning they have difficulty with balance and walking that tends to get worse over time. They also experience pyramidal signs, which are problems with the nerve pathways that control voluntary movement, leading to stiffness and abnormal reflexes. Nystagmus (involuntary, repetitive eye movements) and oculomotor apraxia (difficulty moving the eyes voluntarily to look at specific targets) are also hallmark features of this syndrome. Symptoms typically begin in childhood and progressively worsen. Affected individuals may have trouble walking, speaking clearly, and performing fine motor tasks like writing. The condition follows an autosomal recessive inheritance pattern, meaning a child must inherit a faulty gene copy from each parent to develop the disease. Because this is an extremely rare condition, treatment options are currently limited to managing symptoms and providing supportive care. Physical therapy, occupational therapy, and speech therapy can help maintain function and quality of life. There are no approved disease-modifying treatments at this time, and research into the underlying genetic causes is ongoing.

Key symptoms:

Problems with balance and coordination (ataxia)Unsteady walking that worsens over timeInvoluntary eye movements (nystagmus)Difficulty moving the eyes to look at things on purposeMuscle stiffness or spasticityOveractive reflexesSlurred or unclear speechDifficulty with fine motor tasks like writing or buttoning clothesTremor or shaking during movementProgressive difficulty with walkingPoor hand-eye coordination

Clinical phenotype terms (32)— hover any for plain English
Hypometric saccadesHP:0000571Rotary nystagmusHP:0001583
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 ↗

Treatments

No FDA-approved treatments are currently listed for Autosomal recessive cerebellar ataxia-pyramidal signs-nystagmus-oculomotor apraxia syndrome.

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No specialists are currently listed for Autosomal recessive cerebellar ataxia-pyramidal signs-nystagmus-oculomotor apraxia 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 Autosomal recessive cerebellar ataxia-pyramidal signs-nystagmus-oculomotor apraxia syndrome.

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Community

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Latest news about Autosomal recessive cerebellar ataxia-pyramidal signs-nystagmus-oculomotor apraxia syndrome

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Caregiver Resources

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Questions for your doctor

Bring these to your next appointment

  • Q1.What genetic testing has been done, and are there additional tests that could help identify the exact cause?,How quickly is this condition likely to progress in my case or my child's case?,What therapies do you recommend to help maintain mobility and function?,Are there any clinical trials or research studies we could participate in?,What assistive devices or home modifications should we consider?,How often should we schedule follow-up appointments and with which specialists?,Are there any medications that could help with specific symptoms like stiffness or eye movement problems?

Common questions about Autosomal recessive cerebellar ataxia-pyramidal signs-nystagmus-oculomotor apraxia syndrome

What is Autosomal recessive cerebellar ataxia-pyramidal signs-nystagmus-oculomotor apraxia syndrome?

Autosomal recessive cerebellar ataxia with pyramidal signs, nystagmus, and oculomotor apraxia syndrome is a very rare inherited neurological condition that affects the brain's ability to coordinate movement. The disease primarily impacts the cerebellum, which is the part of the brain responsible for balance and smooth, coordinated movements. People with this condition develop cerebellar ataxia, meaning they have difficulty with balance and walking that tends to get worse over time. They also experience pyramidal signs, which are problems with the nerve pathways that control voluntary movement,

How is Autosomal recessive cerebellar ataxia-pyramidal signs-nystagmus-oculomotor apraxia syndrome inherited?

Autosomal recessive cerebellar ataxia-pyramidal signs-nystagmus-oculomotor apraxia syndrome follows a autosomal recessive inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.

At what age does Autosomal recessive cerebellar ataxia-pyramidal signs-nystagmus-oculomotor apraxia syndrome typically begin?

Typical onset of Autosomal recessive cerebellar ataxia-pyramidal signs-nystagmus-oculomotor apraxia syndrome is childhood. Age of onset can vary across affected individuals.