Brachydactyly-nystagmus-cerebellar ataxia syndrome

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ORPHA:1246OMIM:113400Q87.8
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Overview

Brachydactyly-nystagmus-cerebellar ataxia syndrome is an extremely rare genetic condition that affects multiple body systems. The name describes its three main features: brachydactyly (unusually short fingers and toes), nystagmus (involuntary, repetitive eye movements), and cerebellar ataxia (problems with balance and coordination caused by issues in the cerebellum, the part of the brain that controls movement). People with this syndrome may notice difficulty walking steadily, problems with fine motor tasks like writing or buttoning clothes, and vision difficulties related to the abnormal eye movements. The short fingers and toes are typically present from birth and may vary in severity. This syndrome has been described in only a very small number of families in the medical literature, making it one of the rarest known genetic conditions. Because so few cases have been reported, our understanding of the full range of symptoms, the best treatments, and the long-term outlook is still limited. There is currently no cure for this condition. Treatment focuses on managing individual symptoms, such as physical therapy for coordination problems, occupational therapy for daily tasks, and ophthalmological care for the eye movement disorder. Genetic counseling is recommended for affected families to understand the inheritance pattern and recurrence risk.

Also known as:

Key symptoms:

Short fingers and toesInvoluntary eye movements (eyes moving back and forth uncontrollably)Poor balance and unsteady walkingDifficulty with coordinationTrouble with fine motor skills like writing or picking up small objectsBlurred or unstable visionDifficulty with speech coordinationMild hand and foot abnormalities

Inheritance

Autosomal dominant

Passed on from just one parent; each child has about a 50% chance of inheriting it

Age of Onset

Childhood

Begins in childhood, roughly ages 1 to 12

Orphanet ↗OMIM ↗NORD ↗

Treatments

No FDA-approved treatments are currently listed for Brachydactyly-nystagmus-cerebellar ataxia syndrome.

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No actively recruiting trials found for Brachydactyly-nystagmus-cerebellar ataxia syndrome at this time.

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No specialists are currently listed for Brachydactyly-nystagmus-cerebellar ataxia 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 Brachydactyly-nystagmus-cerebellar ataxia syndrome.

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Community

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Caregiver Resources

NORD Caregiver Resources

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Mental Health Support

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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.How severe is my child's (or my) cerebellar ataxia, and is it likely to get worse over time?,What therapies do you recommend to help with balance and coordination?,Are there any treatments available to reduce the involuntary eye movements?,Should we pursue genetic testing, and what might it tell us?,What accommodations should we request at school or work?,How often should we schedule follow-up appointments with each specialist?,Are there any clinical trials or research studies we could participate in?

Common questions about Brachydactyly-nystagmus-cerebellar ataxia syndrome

What is Brachydactyly-nystagmus-cerebellar ataxia syndrome?

Brachydactyly-nystagmus-cerebellar ataxia syndrome is an extremely rare genetic condition that affects multiple body systems. The name describes its three main features: brachydactyly (unusually short fingers and toes), nystagmus (involuntary, repetitive eye movements), and cerebellar ataxia (problems with balance and coordination caused by issues in the cerebellum, the part of the brain that controls movement). People with this syndrome may notice difficulty walking steadily, problems with fine motor tasks like writing or buttoning clothes, and vision difficulties related to the abnormal eye

How is Brachydactyly-nystagmus-cerebellar ataxia syndrome inherited?

Brachydactyly-nystagmus-cerebellar ataxia syndrome follows a autosomal dominant inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.

At what age does Brachydactyly-nystagmus-cerebellar ataxia syndrome typically begin?

Typical onset of Brachydactyly-nystagmus-cerebellar ataxia syndrome is childhood. Age of onset can vary across affected individuals.