Sensory ataxic neuropathy-dysarthria-ophthalmoparesis syndrome

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ORPHA:70595OMIM:607459G71.3
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8Treatment centers

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Overview

Sensory ataxic neuropathy-dysarthria-ophthalmoparesis syndrome, often called SANDO, is a rare inherited disease that affects the nervous system and muscles. It is caused by problems in the mitochondria — the tiny power plants inside your cells that produce energy. When mitochondria do not work properly, the parts of the body that need the most energy, like nerves and muscles, are hit the hardest. The main features of SANDO include sensory ataxia (difficulty with balance and coordination because the nerves that tell your brain where your body is in space stop working properly), dysarthria (slurred or difficult speech caused by weak muscles in the mouth and throat), and ophthalmoparesis (weakness or paralysis of the eye muscles, making it hard to move the eyes normally). People with SANDO may also experience muscle weakness, hearing loss, and cognitive changes over time. SANDO is part of a group of conditions called mitochondrial DNA depletion syndromes. It is most often caused by changes (mutations) in the POLG gene. There is currently no cure for SANDO, but supportive treatments can help manage symptoms and improve quality of life. The disease tends to progress slowly over many years, and the experience can vary quite a bit from person to person.

Also known as:

Key symptoms:

Poor balance and unsteady walking (sensory ataxia)Slurred or slow speech (dysarthria)Weakness or inability to move the eyes normally (ophthalmoparesis)Drooping eyelids (ptosis)Numbness, tingling, or loss of feeling in the hands and feetMuscle weaknessHearing lossDifficulty swallowingMemory problems or cognitive changesFatigue and low energySeizures in some peopleDepression or mood changes

Clinical phenotype terms (31)— hover any for plain English
Abnormal vestibular functionHP:0001751Positive Romberg signHP:0002403Sensory ataxic neuropathyHP:0003434Impaired distal proprioceptionHP:0006858Atrophy/Degeneration involving the spinal cordHP:0007344Bilateral sensorineural hearing impairmentHP:0008619Abnormal thalamic MRI signal intensityHP:0012696Upgaze palsyHP:0025331
Inheritance

Autosomal recessive

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

Age of Onset

Adult

Begins in adulthood (age 18 or older)

Orphanet ↗OMIM ↗NORD ↗

Treatments

No FDA-approved treatments are currently listed for Sensory ataxic neuropathy-dysarthria-ophthalmoparesis syndrome.

View clinical trials →

No actively recruiting trials found for Sensory ataxic neuropathy-dysarthria-ophthalmoparesis syndrome at this time.

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Search ClinicalTrials.gov ↗Join the Sensory ataxic neuropathy-dysarthria-ophthalmoparesis syndrome community →

No specialists are currently listed for Sensory ataxic neuropathy-dysarthria-ophthalmoparesis 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 Sensory ataxic neuropathy-dysarthria-ophthalmoparesis syndrome.

Search all travel grants →NORD Financial Assistance ↗

Community

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Caregiver Resources

NORD Caregiver Resources

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Social Security Disability

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Questions for your doctor

Bring these to your next appointment

  • Q1.Which specific POLG mutations do I have, and what do they mean for my prognosis?,Are there any medications I should avoid because of my POLG mutation, such as valproic acid?,What therapies — physical, occupational, or speech — do you recommend starting now?,Should other family members be tested for POLG mutations?,Are there any clinical trials for SANDO or POLG-related diseases that I might be eligible for?,How often should I have follow-up appointments, and what monitoring tests do I need regularly?,What signs of worsening should prompt me to seek emergency care?

Common questions about Sensory ataxic neuropathy-dysarthria-ophthalmoparesis syndrome

What is Sensory ataxic neuropathy-dysarthria-ophthalmoparesis syndrome?

Sensory ataxic neuropathy-dysarthria-ophthalmoparesis syndrome, often called SANDO, is a rare inherited disease that affects the nervous system and muscles. It is caused by problems in the mitochondria — the tiny power plants inside your cells that produce energy. When mitochondria do not work properly, the parts of the body that need the most energy, like nerves and muscles, are hit the hardest. The main features of SANDO include sensory ataxia (difficulty with balance and coordination because the nerves that tell your brain where your body is in space stop working properly), dysarthria (slu

How is Sensory ataxic neuropathy-dysarthria-ophthalmoparesis syndrome inherited?

Sensory ataxic neuropathy-dysarthria-ophthalmoparesis syndrome follows a autosomal recessive inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.

At what age does Sensory ataxic neuropathy-dysarthria-ophthalmoparesis syndrome typically begin?

Typical onset of Sensory ataxic neuropathy-dysarthria-ophthalmoparesis syndrome is adult. Age of onset can vary across affected individuals.