Autosomal dominant Charcot-Marie-Tooth disease type 2 due to DGAT2 mutation

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Overview

Autosomal dominant Charcot-Marie-Tooth disease type 2 due to DGAT2 mutation (also called CMT2-DGAT2 or DGAT2-related CMT2) is a rare inherited nerve disease that affects the peripheral nervous system — the network of nerves outside the brain and spinal cord. It belongs to a large family of conditions called Charcot-Marie-Tooth disease (CMT), which are among the most common inherited nerve disorders overall, though this specific subtype caused by changes in the DGAT2 gene is very rare. The disease mainly damages the axons — the long, wire-like parts of nerve cells that carry signals between the brain, spinal cord, muscles, and skin. Over time, this damage leads to muscle weakness and wasting, especially in the feet, lower legs, hands, and forearms. People often notice difficulty walking, foot deformities like high arches or hammertoes, and reduced ability to feel sensations such as touch, pain, or temperature in the hands and feet. There is currently no cure for CMT2-DGAT2. Treatment focuses on managing symptoms and maintaining quality of life. Physical therapy, occupational therapy, orthotic devices such as ankle-foot braces, and pain management are the main tools available. Most people with this condition have a normal life expectancy, but the disease can slowly progress and affect independence and daily activities over time.

Also known as:

Key symptoms:

Muscle weakness in the feet and lower legsMuscle weakness in the hands and forearmsMuscle wasting (shrinking) in the lower legs and feetHigh foot arches (pes cavus)Hammertoes or other foot deformitiesDifficulty walking or frequent tripping and fallsReduced ability to feel touch, pain, or temperature in the feet and handsNumbness or tingling in the hands and feetDecreased reflexes, especially at the anklesFoot drop (difficulty lifting the front part of the foot when walking)

Inheritance

Autosomal dominant

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

Age of Onset

Adult

Begins in adulthood (age 18 or older)

Orphanet ↗NORD ↗

Treatments

No FDA-approved treatments are currently listed for Autosomal dominant Charcot-Marie-Tooth disease type 2 due to DGAT2 mutation.

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No actively recruiting trials found for Autosomal dominant Charcot-Marie-Tooth disease type 2 due to DGAT2 mutation at this time.

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No specialists are currently listed for Autosomal dominant Charcot-Marie-Tooth disease type 2 due to DGAT2 mutation.

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 Autosomal dominant Charcot-Marie-Tooth disease type 2 due to DGAT2 mutation.

Search all travel grants →NORD Financial Assistance ↗

Community

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Latest news about Autosomal dominant Charcot-Marie-Tooth disease type 2 due to DGAT2 mutation

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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

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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.What genetic testing do you recommend to confirm my diagnosis, and should my family members be tested?,How quickly do you expect my symptoms to progress, and what signs should I watch for?,What therapies or devices would help me most right now to stay mobile and independent?,Are there any clinical trials for CMT2 or DGAT2-related disease that I might be eligible for?,What pain management options are available if I develop nerve pain?,Should I see any other specialists, such as an orthopaedic surgeon or rehabilitation doctor?,What lifestyle changes — such as exercise or activity modifications — would be most helpful for me?

Common questions about Autosomal dominant Charcot-Marie-Tooth disease type 2 due to DGAT2 mutation

What is Autosomal dominant Charcot-Marie-Tooth disease type 2 due to DGAT2 mutation?

Autosomal dominant Charcot-Marie-Tooth disease type 2 due to DGAT2 mutation (also called CMT2-DGAT2 or DGAT2-related CMT2) is a rare inherited nerve disease that affects the peripheral nervous system — the network of nerves outside the brain and spinal cord. It belongs to a large family of conditions called Charcot-Marie-Tooth disease (CMT), which are among the most common inherited nerve disorders overall, though this specific subtype caused by changes in the DGAT2 gene is very rare. The disease mainly damages the axons — the long, wire-like parts of nerve cells that carry signals between th

How is Autosomal dominant Charcot-Marie-Tooth disease type 2 due to DGAT2 mutation inherited?

Autosomal dominant Charcot-Marie-Tooth disease type 2 due to DGAT2 mutation follows a autosomal dominant inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.

At what age does Autosomal dominant Charcot-Marie-Tooth disease type 2 due to DGAT2 mutation typically begin?

Typical onset of Autosomal dominant Charcot-Marie-Tooth disease type 2 due to DGAT2 mutation is adult. Age of onset can vary across affected individuals.