Autosomal recessive cerebellar ataxia-psychomotor delay syndrome

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ORPHA:284271OMIM:614229G11.1
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Overview

Autosomal recessive cerebellar ataxia-psychomotor delay syndrome is a rare inherited neurological condition that affects the brain's ability to coordinate movement and mental development. The disease primarily involves the cerebellum, which is the part of the brain responsible for balance, coordination, and smooth movement. Children with this condition typically show delays in reaching developmental milestones such as sitting, walking, and speaking. They may have difficulty with balance and coordination (called ataxia), which can make walking unsteady and fine motor tasks like writing or buttoning clothes challenging. Intellectual disability or learning difficulties of varying severity are also common features. Because this is an autosomal recessive condition, a child must inherit two copies of the faulty gene — one from each parent — to develop the disease. Parents who each carry one copy are typically unaffected. The syndrome usually becomes apparent in infancy or early childhood when developmental delays are first noticed. There is currently no cure for this condition. Treatment focuses on managing symptoms and supporting development through physical therapy, occupational therapy, speech therapy, and educational support. The severity of symptoms can vary between affected individuals, and ongoing medical care from a team of specialists is important to optimize quality of life.

Also known as:

Key symptoms:

Unsteady or wobbly walking (ataxia)Delayed motor milestones such as sitting and walkingDelayed speech developmentIntellectual disability or learning difficultiesPoor coordination of hand and arm movementsLow muscle tone (floppiness)Difficulty with balanceSlow psychomotor developmentTremor or shaking during purposeful movementsDifficulty with fine motor tasks like writing or using utensilsAbnormal eye movements

Clinical phenotype terms (13)— hover any for plain English
Abnormality of ocular smooth pursuitHP:0000617Gaze-evoked horizontal nystagmusHP:0007979
Inheritance

Autosomal recessive

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

Age of Onset

Infantile

Begins in infancy, roughly 1 month to 2 years old

Orphanet ↗OMIM ↗NORD ↗

Treatments

No FDA-approved treatments are currently listed for Autosomal recessive cerebellar ataxia-psychomotor delay syndrome.

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No specialists are currently listed for Autosomal recessive cerebellar ataxia-psychomotor delay 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 Autosomal recessive cerebellar ataxia-psychomotor delay syndrome.

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Community

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Caregiver Resources

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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.What specific genetic mutation is causing my child's condition, and what does it mean for their future?,What therapies do you recommend, and how often should they occur?,Are there any clinical trials or research studies my child might be eligible for?,What developmental milestones should we realistically expect, and on what timeline?,Should other family members be tested for carrier status?,What school accommodations should we request for our child?,Are there any complications we should watch for as our child grows?

Common questions about Autosomal recessive cerebellar ataxia-psychomotor delay syndrome

What is Autosomal recessive cerebellar ataxia-psychomotor delay syndrome?

Autosomal recessive cerebellar ataxia-psychomotor delay syndrome is a rare inherited neurological condition that affects the brain's ability to coordinate movement and mental development. The disease primarily involves the cerebellum, which is the part of the brain responsible for balance, coordination, and smooth movement. Children with this condition typically show delays in reaching developmental milestones such as sitting, walking, and speaking. They may have difficulty with balance and coordination (called ataxia), which can make walking unsteady and fine motor tasks like writing or butto

How is Autosomal recessive cerebellar ataxia-psychomotor delay syndrome inherited?

Autosomal recessive cerebellar ataxia-psychomotor delay 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-psychomotor delay syndrome typically begin?

Typical onset of Autosomal recessive cerebellar ataxia-psychomotor delay syndrome is infantile. Age of onset can vary across affected individuals.