Laminin subunit alpha 2-related limb-girdle muscular dystrophy R23

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ORPHA:565837OMIM:618138G71.0
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Overview

Laminin subunit alpha 2-related limb-girdle muscular dystrophy R23, also called LGMD R23 or LAMA2-related LGMD, is a rare inherited muscle disease caused by changes (variants) in the LAMA2 gene. This gene provides instructions for making a protein called laminin alpha-2, which is an important building block that helps keep muscle fibers strong and stable. When this protein is missing or does not work properly, muscle fibers become fragile and break down over time. This condition mainly affects the muscles closest to the center of the body — the hips, thighs, and shoulders — which is why it is called 'limb-girdle' muscular dystrophy. People with LGMD R23 typically notice progressive muscle weakness that makes it harder to walk, climb stairs, lift objects, or get up from the floor. The condition is different from the more severe LAMA2-related congenital muscular dystrophy (MDC1A), which starts at birth and is usually more serious. LGMD R23 tends to begin later and progress more slowly, though this can vary from person to person. There is currently no cure for LGMD R23. Treatment focuses on managing symptoms, maintaining strength and mobility through physical therapy, and monitoring for complications such as breathing or heart problems. A team of specialists works together to help people live as well as possible with this condition.

Also known as:

Key symptoms:

Weakness in the hip and thigh musclesWeakness in the shoulder and upper arm musclesDifficulty walking or waddling gaitTrouble climbing stairs or getting up from the floorDifficulty lifting arms above the headMuscle wasting (muscles getting smaller over time)Fatigue with physical activityPossible shortness of breath if breathing muscles are affectedPossible heart muscle involvement (cardiomyopathy) in some casesElevated creatine kinase (CK) levels in the blood, a sign of muscle damage

Inheritance

Autosomal recessive

Passed on when both parents carry the same gene change; often skips generations

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 Laminin subunit alpha 2-related limb-girdle muscular dystrophy R23.

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No actively recruiting trials found for Laminin subunit alpha 2-related limb-girdle muscular dystrophy R23 at this time.

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No specialists are currently listed for Laminin subunit alpha 2-related limb-girdle muscular dystrophy R23.

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 Laminin subunit alpha 2-related limb-girdle muscular dystrophy R23.

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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 severe is my (or my child's) LAMA2 gene change, and what does that mean for how the disease might progress?,How often should I have heart and lung function checked, and what warning signs should prompt me to seek care sooner?,What type of physical therapy is most appropriate, and are there any exercises I should avoid?,Are there any clinical trials for LAMA2-related muscular dystrophy that I might be eligible for?,Should other family members be tested for this condition?,What assistive devices or home modifications might help me maintain independence?,What specialists should be part of my care team, and how do I coordinate care between them?

Common questions about Laminin subunit alpha 2-related limb-girdle muscular dystrophy R23

What is Laminin subunit alpha 2-related limb-girdle muscular dystrophy R23?

Laminin subunit alpha 2-related limb-girdle muscular dystrophy R23, also called LGMD R23 or LAMA2-related LGMD, is a rare inherited muscle disease caused by changes (variants) in the LAMA2 gene. This gene provides instructions for making a protein called laminin alpha-2, which is an important building block that helps keep muscle fibers strong and stable. When this protein is missing or does not work properly, muscle fibers become fragile and break down over time. This condition mainly affects the muscles closest to the center of the body — the hips, thighs, and shoulders — which is why it is

How is Laminin subunit alpha 2-related limb-girdle muscular dystrophy R23 inherited?

Laminin subunit alpha 2-related limb-girdle muscular dystrophy R23 follows a autosomal recessive inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.