Severe motor and intellectual disabilities-sensorineural deafness-dystonia syndrome

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ORPHA:369939OMIM:300475Q87.8
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Overview

Severe motor and intellectual disabilities-sensorineural deafness-dystonia syndrome is an extremely rare genetic condition that affects multiple body systems, particularly the brain and nervous system. The disease is characterized by a combination of serious movement problems (severe motor disability), significant learning and thinking difficulties (intellectual disability), hearing loss caused by nerve damage in the inner ear (sensorineural deafness), and dystonia, which involves involuntary muscle contractions that cause abnormal postures or twisting movements. This condition typically becomes apparent very early in life. Affected children show profound delays in reaching developmental milestones such as sitting, standing, and walking. Many individuals are unable to walk independently or communicate verbally. The hearing loss can range from moderate to profound and further complicates communication and learning. Dystonia can affect various parts of the body and may cause significant discomfort and difficulty with daily activities. Because this syndrome is so rare, treatment options are currently limited to managing individual symptoms. There is no cure available at this time. Care typically involves a team of specialists working together to address the movement difficulties, hearing loss, and developmental needs of the affected individual. Supportive therapies such as physical therapy, occupational therapy, speech therapy, and hearing aids or cochlear implants may be recommended to improve quality of life.

Also known as:

Key symptoms:

Severe intellectual disabilityInability to walk or severe difficulty with movementHearing loss present from birth or early infancyInvoluntary muscle contractions causing abnormal postures (dystonia)Severe speech and language delays or absence of speechDifficulty feeding or swallowingLow muscle tone in early lifeDelayed developmental milestonesSeizures in some casesAbnormal eye movementsPoor head controlLimited ability to use hands purposefullyGrowth delays

Clinical phenotype terms (20)— hover any for plain English
TetraplegiaHP:0002445Cerebral hypomyelinationHP:0006808CNS hypomyelinationHP:0003429Corpus callosum atrophyHP:0007371Cerebral white matter atrophyHP:0012762
Inheritance

Autosomal recessive

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

Age of Onset

Neonatal

Begins at or shortly after birth (first 4 weeks)

Orphanet ↗OMIM ↗NORD ↗

Treatments

No FDA-approved treatments are currently listed for Severe motor and intellectual disabilities-sensorineural deafness-dystonia syndrome.

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No actively recruiting trials found for Severe motor and intellectual disabilities-sensorineural deafness-dystonia syndrome at this time.

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No specialists are currently listed for Severe motor and intellectual disabilities-sensorineural deafness-dystonia 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 Severe motor and intellectual disabilities-sensorineural deafness-dystonia syndrome.

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Community

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Caregiver Resources

NORD Caregiver Resources

Support, advocacy, and financial assistance for caregivers of rare disease patients.

Mental Health Support

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Family & Caregiver Grants

Financial assistance programs specifically for caregivers of rare disease patients.

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 does my child have, and what does it mean for their condition?,What therapies should we start right away to give my child the best chance at development?,Would hearing aids or cochlear implants be appropriate for my child's type of hearing loss?,What medications can help manage the dystonia, and what are the side effects?,Are there any clinical trials or research studies we should know about?,What is the risk of having another child with this condition, and should we pursue genetic counseling?,What emergency signs should we watch for at home, and when should we go to the hospital?

Common questions about Severe motor and intellectual disabilities-sensorineural deafness-dystonia syndrome

What is Severe motor and intellectual disabilities-sensorineural deafness-dystonia syndrome?

Severe motor and intellectual disabilities-sensorineural deafness-dystonia syndrome is an extremely rare genetic condition that affects multiple body systems, particularly the brain and nervous system. The disease is characterized by a combination of serious movement problems (severe motor disability), significant learning and thinking difficulties (intellectual disability), hearing loss caused by nerve damage in the inner ear (sensorineural deafness), and dystonia, which involves involuntary muscle contractions that cause abnormal postures or twisting movements. This condition typically beco

How is Severe motor and intellectual disabilities-sensorineural deafness-dystonia syndrome inherited?

Severe motor and intellectual disabilities-sensorineural deafness-dystonia syndrome follows a autosomal recessive inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.

At what age does Severe motor and intellectual disabilities-sensorineural deafness-dystonia syndrome typically begin?

Typical onset of Severe motor and intellectual disabilities-sensorineural deafness-dystonia syndrome is neonatal. Age of onset can vary across affected individuals.