Distal myopathy with early respiratory muscle involvement

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Overview

Distal myopathy with early respiratory muscle involvement (also sometimes called DMRV or Nonaka myopathy in some overlapping contexts, though this specific form is distinct) is a rare inherited muscle disease. It mainly affects the muscles farthest from the center of the body — like those in the hands, feet, lower legs, and forearms — causing progressive weakness in these areas. What makes this condition especially unusual compared to other distal myopathies is that the breathing muscles become weak relatively early in the disease course, which is not typical for most muscle diseases that start in the hands and feet. People with this condition usually notice weakness in their legs or feet first, which can cause tripping, foot drop, or difficulty walking. Over time, weakness can spread to other muscle groups. Because the respiratory muscles are affected early, breathing problems can develop even before mobility becomes severely limited. This means regular monitoring of lung function is a critical part of care. There is currently no cure for this condition. Treatment focuses on managing symptoms, supporting breathing with devices like non-invasive ventilators when needed, physical therapy to maintain strength and mobility, and regular monitoring by a team of specialists. With careful management — especially attention to respiratory health — many people can maintain a good quality of life for many years after diagnosis.

Key symptoms:

Weakness in the lower legs and feetFoot drop — difficulty lifting the front part of the foot when walkingTripping or frequent fallsWeakness in the hands and forearmsShortness of breath, especially during activity or when lying flatDifficulty breathing during sleepReduced exercise tolerance and easy fatigueWasting (shrinking) of muscles in the lower legs and handsDifficulty climbing stairsWeak grip strength

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 Distal myopathy with early respiratory muscle involvement.

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No actively recruiting trials found for Distal myopathy with early respiratory muscle involvement at this time.

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Search ClinicalTrials.gov ↗Join the Distal myopathy with early respiratory muscle involvement community →

No specialists are currently listed for Distal myopathy with early respiratory muscle involvement.

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 Distal myopathy with early respiratory muscle involvement.

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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.How quickly do you expect my symptoms to progress, and what signs should I watch for?,How often should I have my breathing (lung function) tested, and at what point would I need breathing support?,Should other members of my family be tested for this condition?,Are there any clinical trials I might be eligible for?,What physical therapy or exercise program is safe and beneficial for me?,What assistive devices or home modifications would you recommend now or in the future?,Who should I contact if I develop sudden breathing difficulties?

Common questions about Distal myopathy with early respiratory muscle involvement

What is Distal myopathy with early respiratory muscle involvement?

Distal myopathy with early respiratory muscle involvement (also sometimes called DMRV or Nonaka myopathy in some overlapping contexts, though this specific form is distinct) is a rare inherited muscle disease. It mainly affects the muscles farthest from the center of the body — like those in the hands, feet, lower legs, and forearms — causing progressive weakness in these areas. What makes this condition especially unusual compared to other distal myopathies is that the breathing muscles become weak relatively early in the disease course, which is not typical for most muscle diseases that star

How is Distal myopathy with early respiratory muscle involvement inherited?

Distal myopathy with early respiratory muscle involvement follows a autosomal dominant inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.

At what age does Distal myopathy with early respiratory muscle involvement typically begin?

Typical onset of Distal myopathy with early respiratory muscle involvement is adult. Age of onset can vary across affected individuals.