Mohr-Tranebjaerg syndrome

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ORPHA:52368OMIM:304700E88.8
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Overview

Mohr-Tranebjaerg syndrome (MTS) is a very rare inherited condition that mainly affects hearing and the nervous system. It is also known as deafness-dystonia-optic neuronopathy (DDON) syndrome. The condition is caused by a change (mutation) in a gene called TIMM8A, which sits on the X chromosome. Because of this, it mostly affects males, while females who carry the gene change usually have milder symptoms or none at all. The most noticeable early sign is progressive hearing loss that usually begins in childhood. Over time, many people with MTS also develop problems with movement control (called dystonia), which can cause involuntary muscle contractions and awkward postures. Vision loss due to damage to the optic nerve (the nerve connecting the eye to the brain) often appears later in life. Some individuals also experience changes in behavior or thinking, and in some cases, a condition similar to Parkinson's disease can develop in adulthood. There is currently no cure for Mohr-Tranebjaerg syndrome. Treatment focuses on managing symptoms. Hearing aids or cochlear implants can help with hearing loss. Physical therapy, occupational therapy, and medications may help manage movement problems. A team of specialists is usually needed to provide the best care. Early diagnosis is important so that supportive therapies can be started as soon as possible.

Also known as:

Key symptoms:

Progressive hearing loss starting in childhoodInvoluntary muscle contractions or abnormal postures (dystonia)Gradual vision loss due to optic nerve damageDifficulty with balance and coordinationChanges in behavior or personalityDecline in thinking or memory in some individualsSwallowing difficulties in some casesParkinson's-like symptoms (tremor, stiffness, slow movement) in some adultsAnxiety or psychiatric symptoms in some individuals

Clinical phenotype terms (41)— hover any for plain English
Abnormal cochlea morphologyHP:0000375Prelingual sensorineural hearing impairmentHP:0000399Abnormal vestibular functionHP:0001751Global brain atrophyHP:0002283Absent brainstem auditory responsesHP:0004463Hyperactive deep tendon reflexesHP:0006801Generalized dystoniaHP:0007325Abnormality of somatosensory evoked potentialsHP:0007377Postlingual sensorineural hearing impairmentHP:0008596Ankle clonusHP:0011448Oromandibular dystoniaHP:0012048
Inheritance

X-linked recessive

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

Age of Onset

Childhood

Begins in childhood, roughly ages 1 to 12

Orphanet ↗OMIM ↗NORD ↗

Treatments

No FDA-approved treatments are currently listed for Mohr-Tranebjaerg syndrome.

View clinical trials →

No actively recruiting trials found for Mohr-Tranebjaerg syndrome at this time.

New trials open frequently. Follow this disease to get notified.

Search ClinicalTrials.gov ↗Join the Mohr-Tranebjaerg syndrome community →

No specialists are currently listed for Mohr-Tranebjaerg 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 Mohr-Tranebjaerg syndrome.

Search all travel grants →NORD Financial Assistance ↗

Community

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Caregiver Resources

NORD Caregiver Resources

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

Mental Health Support

Rare disease caregiving can be isolating. Connect with counseling and peer support.

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 genetic testing should my family members have, and what does a positive carrier result mean for female relatives?,Would a cochlear implant be appropriate for my level of hearing loss, and how soon should we consider it?,What therapies are available to help manage my movement problems, and when should we start them?,How often should I have my vision checked, and what signs of worsening should I watch for at home?,Are there any clinical trials or research studies I could participate in?,What support services or educational accommodations are available for someone with this condition?,What should I do if symptoms suddenly get worse — who should I contact first?

Common questions about Mohr-Tranebjaerg syndrome

What is Mohr-Tranebjaerg syndrome?

Mohr-Tranebjaerg syndrome (MTS) is a very rare inherited condition that mainly affects hearing and the nervous system. It is also known as deafness-dystonia-optic neuronopathy (DDON) syndrome. The condition is caused by a change (mutation) in a gene called TIMM8A, which sits on the X chromosome. Because of this, it mostly affects males, while females who carry the gene change usually have milder symptoms or none at all. The most noticeable early sign is progressive hearing loss that usually begins in childhood. Over time, many people with MTS also develop problems with movement control (calle

How is Mohr-Tranebjaerg syndrome inherited?

Mohr-Tranebjaerg syndrome follows a x-linked recessive inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.

At what age does Mohr-Tranebjaerg syndrome typically begin?

Typical onset of Mohr-Tranebjaerg syndrome is childhood. Age of onset can vary across affected individuals.