X-linked scapuloperoneal muscular dystrophy

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ORPHA:431272OMIM:300695G71.0
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Overview

X-linked scapuloperoneal muscular dystrophy is a rare inherited muscle disease that mainly affects males. It belongs to a group of conditions called muscular dystrophies, which cause the muscles to gradually weaken and waste away over time. The name describes where the weakness tends to show up first: the shoulder blade area (scapular) and the lower legs and feet (peroneal). This condition is sometimes referred to as X-linked scapuloperoneal myopathy or scapuloperoneal syndrome, X-linked type. The disease is caused by changes in a gene on the X chromosome, which is why it mostly affects males. Females who carry one changed copy of the gene may have mild symptoms or none at all. The muscle weakness usually starts in the shoulder girdle, making it hard to lift the arms above the head, and in the lower legs, causing foot drop and difficulty walking. Over time, weakness can spread to other muscle groups. There is currently no cure for this condition. Treatment focuses on managing symptoms, maintaining mobility, and improving quality of life. Physical therapy, occupational therapy, and assistive devices like ankle-foot orthoses (braces) are the main tools used. A team of specialists helps monitor breathing, heart function, and overall muscle health as the disease progresses.

Also known as:

Key symptoms:

Weakness in the shoulder blade muscles, making it hard to raise the armsWinging of the shoulder blades (they stick out from the back)Weakness in the lower legs and feetFoot drop (difficulty lifting the front part of the foot when walking)Difficulty walking or a changed walking patternMuscle wasting in the shoulders and lower legsFatigue with physical activityDifficulty climbing stairsPossible mild heart muscle involvement in some casesPossible breathing difficulties in advanced stages

Inheritance

X-linked recessive

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

Age of Onset

Adult

Begins in adulthood (age 18 or older)

Orphanet ↗OMIM ↗NORD ↗

Treatments

No FDA-approved treatments are currently listed for X-linked scapuloperoneal muscular dystrophy.

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No actively recruiting trials found for X-linked scapuloperoneal muscular dystrophy at this time.

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Search ClinicalTrials.gov ↗Join the X-linked scapuloperoneal muscular dystrophy community →

No specialists are currently listed for X-linked scapuloperoneal muscular dystrophy.

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 X-linked scapuloperoneal muscular dystrophy.

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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.Which specific gene change do I have, and what does that mean for how my disease might progress?,Should my heart and lungs be tested now, and how often should they be monitored?,What physical therapy or exercises are safe and helpful for me at this stage?,Are there any clinical trials I might be eligible for?,Should my female relatives be tested to see if they are carriers?,What signs should prompt me to seek emergency care?,Are there any assistive devices or home modifications that could help me stay independent longer?

Common questions about X-linked scapuloperoneal muscular dystrophy

What is X-linked scapuloperoneal muscular dystrophy?

X-linked scapuloperoneal muscular dystrophy is a rare inherited muscle disease that mainly affects males. It belongs to a group of conditions called muscular dystrophies, which cause the muscles to gradually weaken and waste away over time. The name describes where the weakness tends to show up first: the shoulder blade area (scapular) and the lower legs and feet (peroneal). This condition is sometimes referred to as X-linked scapuloperoneal myopathy or scapuloperoneal syndrome, X-linked type. The disease is caused by changes in a gene on the X chromosome, which is why it mostly affects males

How is X-linked scapuloperoneal muscular dystrophy inherited?

X-linked scapuloperoneal muscular dystrophy follows a x-linked recessive inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.

At what age does X-linked scapuloperoneal muscular dystrophy typically begin?

Typical onset of X-linked scapuloperoneal muscular dystrophy is adult. Age of onset can vary across affected individuals.