X-linked non progressive cerebellar ataxia

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ORPHA:314978OMIM:300703G11.0
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Overview

X-linked non-progressive cerebellar ataxia is a rare genetic condition that affects coordination and balance. It is sometimes also called X-linked congenital cerebellar hypoplasia or X-linked cerebellar ataxia. The condition is caused by changes in genes located on the X chromosome, which means it primarily affects boys and men, while females are usually carriers who may have mild or no symptoms. The cerebellum is the part of the brain responsible for coordinating movement, balance, and fine motor skills. In this condition, the cerebellum does not develop fully or function properly, leading to problems with coordination (ataxia) from early in life. Importantly, the word 'non-progressive' means that the condition does not get worse over time, which distinguishes it from many other forms of ataxia. Key symptoms include unsteady walking, poor coordination of hand and arm movements, difficulty with balance, and sometimes mild intellectual disability or speech difficulties. Symptoms are usually noticed in infancy or early childhood when a child is learning to walk and move. There is currently no cure for this condition, and treatment focuses on supportive therapies such as physical therapy, occupational therapy, and speech therapy to help individuals maximize their abilities and independence. Some individuals may also benefit from assistive devices for mobility.

Key symptoms:

Unsteady or wobbly walkingPoor balanceDifficulty coordinating hand and arm movementsDelayed motor milestones such as sitting and walkingSlurred or slow speechTremor or shaking during purposeful movementsLow muscle tone in infancyMild intellectual disability in some casesDifficulty with fine motor tasks like writing or buttoning clothesAbnormal eye movements such as nystagmusWide-based gait

Clinical phenotype terms (16)— hover any for plain English
Nonprogressive cerebellar ataxiaHP:0002470Saccadic smooth pursuit interruptionsHP:0001152Action tremorHP:0002345Generalized neonatal hypotoniaHP:0008935
Inheritance

X-linked recessive

Carried on the X chromosome; typically affects males more than females

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 X-linked non progressive cerebellar ataxia.

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No actively recruiting trials found for X-linked non progressive cerebellar ataxia at this time.

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No specialists are currently listed for X-linked non progressive cerebellar ataxia.

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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 X-linked non progressive cerebellar ataxia.

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Community

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Caregiver Resources

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Social Security Disability

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Questions for your doctor

Bring these to your next appointment

  • Q1.What specific gene change is causing my child's condition, and what does that mean for their outlook?,How often should my child have physical therapy, occupational therapy, and speech therapy?,Are there any clinical trials or new treatments being studied for this condition?,What school accommodations should I request for my child?,Should other family members be tested to see if they are carriers?,Will my child be able to walk independently, and what can we do to support that?,How will this condition affect my child's learning and intellectual development?

Common questions about X-linked non progressive cerebellar ataxia

What is X-linked non progressive cerebellar ataxia?

X-linked non-progressive cerebellar ataxia is a rare genetic condition that affects coordination and balance. It is sometimes also called X-linked congenital cerebellar hypoplasia or X-linked cerebellar ataxia. The condition is caused by changes in genes located on the X chromosome, which means it primarily affects boys and men, while females are usually carriers who may have mild or no symptoms. The cerebellum is the part of the brain responsible for coordinating movement, balance, and fine motor skills. In this condition, the cerebellum does not develop fully or function properly, leading t

How is X-linked non progressive cerebellar ataxia inherited?

X-linked non progressive cerebellar ataxia follows a x-linked recessive inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.

At what age does X-linked non progressive cerebellar ataxia typically begin?

Typical onset of X-linked non progressive cerebellar ataxia is infantile. Age of onset can vary across affected individuals.