Demyelinating hereditary motor and sensory neuropathy

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ORPHA:476116
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21Specialists8Treatment centers

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Overview

Demyelinating hereditary motor and sensory neuropathy (also known as demyelinating HMSN, or a form of Charcot-Marie-Tooth disease type 1, CMT1) is a rare inherited nerve disease that affects the protective covering of nerves called myelin. Myelin works like insulation around an electrical wire — when it breaks down, nerve signals slow down or stop working properly. This disease affects both the motor nerves (which control muscle movement) and the sensory nerves (which carry feelings like touch, pain, and temperature). People with this condition usually notice weakness and wasting of muscles, especially in the feet, legs, and hands. They may also have reduced ability to feel sensations, balance problems, and changes in the shape of their feet such as high arches or hammertoes. Symptoms often begin in childhood or early adulthood and slowly get worse over time. There is currently no cure for demyelinating HMSN, but many supportive treatments can help manage symptoms and improve quality of life. Physical therapy, occupational therapy, orthotics (special shoe inserts or braces), and pain management are the main tools used today. With good care, many people live full and active lives, though some may need mobility aids as the disease progresses.

Also known as:

Key symptoms:

Muscle weakness in the feet and lower legsMuscle weakness in the hands and forearmsLoss of muscle bulk (muscle wasting) in legs and handsReduced ability to feel touch, pain, or temperature in feet and handsHigh arches in the feet (pes cavus)Hammertoes or other foot deformitiesDifficulty walking or frequent tripping and fallingFoot drop (difficulty lifting the front part of the foot)Balance and coordination problemsReduced or absent reflexes (especially at the ankle)Fatigue with physical activityMild to moderate pain or cramping in the legsSlow nerve conduction speeds on nerve tests

Inheritance

Variable

Can be inherited in different ways depending on the underlying gene

Age of Onset

Variable

Can begin at different ages, from infancy through adulthood

Orphanet ↗NORD ↗

Treatments

No FDA-approved treatments are currently listed for Demyelinating hereditary motor and sensory neuropathy.

View clinical trials →

No actively recruiting trials found for Demyelinating hereditary motor and sensory neuropathy at this time.

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Search ClinicalTrials.gov ↗Join the Demyelinating hereditary motor and sensory neuropathy community →

Specialists

21 foundView all specialists →
AM
Adeline Vanderver, MD
Los Angeles, California
Specialist

Rare Disease Specialist

PI on 4 active trials1 Demyelinating hereditary motor and sensory neuropathy publication
JP
Jeremy Shefner, MD, PhD
PHOENIX, AZ
Specialist
PI on 3 active trials
FT
Frédéric TAITHE
Specialist
PI on 2 active trials2 Demyelinating hereditary motor and sensory neuropathy publications
JM
Jakub M Antczak, MD
Krakow, Lesser Poland Voivodeship
Specialist

Rare Disease Specialist

PI on 3 active trials
AM
Alain CREANGE, MD
Specialist
PI on 1 active trial1 Demyelinating hereditary motor and sensory neuropathy publication
VS
Victoria Singh-Curry
Specialist
PI on 1 active trial
LZ
Liqun Zhang
STONY BROOK, NY
Specialist
PI on 1 active trial994 Demyelinating hereditary motor and sensory neuropathy publications
KS
Klaus Schmierer
Specialist
PI on 1 active trial
JP
Joachim Burman, MD, PhD
Specialist
PI on 1 active trial
AM
Alan Hand, MD
SUGAR LAND, TX
Specialist
PI on 1 active trial1 Demyelinating hereditary motor and sensory neuropathy publication
JP
Joep Killestein, MD, PhD.
Specialist
PI on 1 active trial
NP
Nur Yuceyar, Professor
Specialist
PI on 1 active trial
YP
Yann Pereon, PU-PH
Specialist
PI on 1 active trial
CH
Chris Harding
COTTONWOOD HEIGHTS, UT
Specialist
PI on 1 active trial5 Demyelinating hereditary motor and sensory neuropathy publications
MD
Mehmet ACIK, Dr
Specialist
PI on 1 active trial
SP
Seckin Senisik, Ass. Prof.
Specialist
PI on 1 active trial

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 Demyelinating hereditary motor and sensory neuropathy.

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Community

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Caregiver Resources

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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.Which specific gene is causing my condition, and what does that mean for how my disease will progress?,Should other family members be tested, and what are the chances of passing this on to my children?,What therapies or exercises are most important for me to start right now?,Are there any medications I should avoid because they could make my nerve damage worse?,Are there any clinical trials or research studies I might be eligible to join?,How often should I have follow-up nerve conduction studies or other monitoring tests?,What signs should prompt me to seek urgent medical attention?

Common questions about Demyelinating hereditary motor and sensory neuropathy

What is Demyelinating hereditary motor and sensory neuropathy?

Demyelinating hereditary motor and sensory neuropathy (also known as demyelinating HMSN, or a form of Charcot-Marie-Tooth disease type 1, CMT1) is a rare inherited nerve disease that affects the protective covering of nerves called myelin. Myelin works like insulation around an electrical wire — when it breaks down, nerve signals slow down or stop working properly. This disease affects both the motor nerves (which control muscle movement) and the sensory nerves (which carry feelings like touch, pain, and temperature). People with this condition usually notice weakness and wasting of muscles,

Which specialists treat Demyelinating hereditary motor and sensory neuropathy?

21 specialists and care centers treating Demyelinating hereditary motor and sensory neuropathy are listed on UniteRare, sourced from ClinicalTrials.gov principal investigators, published research, and the NPPES NPI registry.