Autosomal dominant limb-girdle muscular dystrophy type 1C

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Overview

Autosomal dominant limb-girdle muscular dystrophy type 1C (LGMD1C), also known as caveolinopathy or caveolin-3 deficiency, is a rare inherited muscle disease. It belongs to a group of conditions called limb-girdle muscular dystrophies, which mainly affect the muscles closest to the body — the hips, thighs, shoulders, and upper arms. In LGMD1C, a problem in the CAV3 gene causes a shortage of a protein called caveolin-3, which is essential for keeping muscle cells healthy and working properly. The main symptoms include muscle weakness and wasting in the hip and shoulder areas, muscle cramps, and sometimes an unusual rippling movement of the muscles that can be triggered by touch or exercise — a feature called rippling muscle disease. Some people also have mildly elevated levels of a muscle enzyme called creatine kinase in their blood, which signals muscle damage. Symptoms usually begin in childhood or early adulthood, though the age of onset can vary. There is currently no cure for LGMD1C. Treatment focuses on managing symptoms, maintaining strength and mobility through physical therapy, and monitoring for complications. With the right support, many people with LGMD1C can maintain a good quality of life, though the disease can slowly progress over time. Genetic counseling is strongly recommended for affected individuals and their families.

Also known as:

Key symptoms:

Muscle weakness in the hips and thighsMuscle weakness in the shoulders and upper armsMuscle cramps, especially during or after exerciseRippling or wave-like movements of the muscles when touched or after exerciseMuscle stiffnessDifficulty climbing stairs or rising from a chairTrouble lifting arms above the headMild to moderately elevated creatine kinase (a blood marker of muscle damage)Muscle wasting over timeExercise intolerance or easy fatigue with physical activity

Inheritance

Autosomal dominant

Passed on from just one parent; each child has about a 50% chance of inheriting it

Age of Onset

Variable

Can begin at different ages, from infancy through adulthood

Orphanet ↗NORD ↗

Treatments

No FDA-approved treatments are currently listed for Autosomal dominant limb-girdle muscular dystrophy type 1C.

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No actively recruiting trials found for Autosomal dominant limb-girdle muscular dystrophy type 1C at this time.

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No specialists are currently listed for Autosomal dominant limb-girdle muscular dystrophy type 1C.

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 Autosomal dominant limb-girdle muscular dystrophy type 1C.

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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.What genetic test should I have, and will my family members need testing too?,How quickly might my muscle weakness progress, and what signs should I watch for?,What type and amount of exercise is safe for me, and what should I avoid?,Should I have my heart checked regularly, and how often?,Are there any medications or supplements I should avoid because they could harm my muscles?,What physical therapy or rehabilitation program would you recommend for me?,Are there any clinical trials or research studies I might be eligible to join?

Common questions about Autosomal dominant limb-girdle muscular dystrophy type 1C

What is Autosomal dominant limb-girdle muscular dystrophy type 1C?

Autosomal dominant limb-girdle muscular dystrophy type 1C (LGMD1C), also known as caveolinopathy or caveolin-3 deficiency, is a rare inherited muscle disease. It belongs to a group of conditions called limb-girdle muscular dystrophies, which mainly affect the muscles closest to the body — the hips, thighs, shoulders, and upper arms. In LGMD1C, a problem in the CAV3 gene causes a shortage of a protein called caveolin-3, which is essential for keeping muscle cells healthy and working properly. The main symptoms include muscle weakness and wasting in the hip and shoulder areas, muscle cramps, an

How is Autosomal dominant limb-girdle muscular dystrophy type 1C inherited?

Autosomal dominant limb-girdle muscular dystrophy type 1C follows a autosomal dominant inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.