Myosin storage myopathy

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ORPHA:53698OMIM:255160G71.2
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1Specialists8Treatment centers

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Overview

Myosin storage myopathy (also called hyaline body myopathy) is a rare inherited muscle disease caused by changes in a gene that makes a protein called myosin, which is essential for muscle movement. In this condition, abnormal clumps of myosin build up inside muscle fibers, forming structures called hyaline bodies. These deposits damage the muscle cells over time and prevent them from working properly. The disease mainly affects the skeletal muscles — the muscles you use to move your body. Most people experience slowly progressive muscle weakness, especially in the shoulders, upper arms, and hips. Some individuals also develop breathing difficulties because the muscles that support the lungs can be affected. Heart muscle involvement has been reported in some cases, though it is less common. Symptoms can appear at any age, from infancy to adulthood, and the severity varies widely even within the same family. There is currently no cure for myosin storage myopathy. Treatment focuses on managing symptoms, maintaining mobility through physical therapy, and monitoring breathing and heart function. With careful management, many people can maintain a good quality of life for many years.

Also known as:

Key symptoms:

Slowly worsening muscle weakness, especially in the shoulders and hipsDifficulty lifting arms above the headTrouble climbing stairs or rising from a chairWasting (shrinking) of affected muscles over timeShortness of breath or difficulty breathing, especially when lying downFatigue with everyday activitiesDrooping of the eyelids (ptosis) in some casesDifficulty swallowing in some casesCurved spine (scoliosis) in some individualsFoot deformities such as high archesHeart rhythm problems or weakened heart muscle in some casesReduced exercise tolerance

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 ↗OMIM ↗NORD ↗

Treatments

No FDA-approved treatments are currently listed for Myosin storage myopathy.

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No actively recruiting trials found for Myosin storage myopathy at this time.

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Specialists

1 foundView all specialists →
NM
Niklas Darin, M.D
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 Myosin storage myopathy.

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Community

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Latest news about Myosin storage myopathy

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Caregiver Resources

NORD Caregiver Resources

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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.Which muscles are most likely to be affected in my case, and how quickly might the weakness progress?,Do I need breathing tests or heart monitoring, and how often should these be done?,Should my family members be tested for the MYH7 gene change?,What physical therapy program would be most helpful for me right now?,Are there any clinical trials or research studies I might be eligible for?,At what point might I need breathing support, and what does that look like?,Are there any activities or exercises I should avoid to protect my muscles?

Common questions about Myosin storage myopathy

What is Myosin storage myopathy?

Myosin storage myopathy (also called hyaline body myopathy) is a rare inherited muscle disease caused by changes in a gene that makes a protein called myosin, which is essential for muscle movement. In this condition, abnormal clumps of myosin build up inside muscle fibers, forming structures called hyaline bodies. These deposits damage the muscle cells over time and prevent them from working properly. The disease mainly affects the skeletal muscles — the muscles you use to move your body. Most people experience slowly progressive muscle weakness, especially in the shoulders, upper arms, and

Which specialists treat Myosin storage myopathy?

1 specialists and care centers treating Myosin storage myopathy are listed on UniteRare, sourced from ClinicalTrials.gov principal investigators, published research, and the NPPES NPI registry.