Delta-sarcoglycan-related limb-girdle muscular dystrophy R6

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ORPHA:219OMIM:601287G71.0
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Overview

Delta-sarcoglycan-related limb-girdle muscular dystrophy R6 (also called LGMD R6 or LGMD2F) is a rare inherited muscle disease caused by changes in the SGCD gene. This gene provides instructions for making a protein called delta-sarcoglycan, which is an important building block of a complex that protects muscle fibers during movement. When this protein is missing or not working properly, muscle fibers become fragile and break down over time. The disease mainly affects the muscles around the hips, thighs, and shoulders — the so-called 'limb-girdle' muscles. People with LGMD R6 typically notice weakness in these areas, which can make it hard to climb stairs, rise from a chair, lift objects overhead, or keep up with physical activities. The heart muscle can also be affected in some people, which is an important concern that needs regular monitoring. Right now, there is no cure for LGMD R6. Treatment focuses on managing symptoms, keeping muscles as strong and flexible as possible through physical therapy, and watching closely for heart or breathing problems. A team of specialists — including neurologists, cardiologists, and physiotherapists — work 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 climbing stairs or getting up from the floorTrouble lifting arms above the headWaddling or unusual walking patternFrequent fallsMuscle wasting (muscles getting smaller over time)Calf muscles that look enlarged but are actually weakHeart muscle problems (cardiomyopathy)Shortness of breath with activityFatigue with everyday tasksPossible breathing difficulties as the disease progresses

Clinical phenotype terms (6)— hover any for plain English
Shuffling gaitHP:0002362Proximal upper limb amyotrophyHP:0008948Proximal lower limb amyotrophyHP:0008956Generalized limb muscle atrophyHP:0009055
Inheritance

Autosomal recessive

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

Age of Onset

Childhood

Begins in childhood, roughly ages 1 to 12

Orphanet ↗OMIM ↗NORD ↗

Treatments

No FDA-approved treatments are currently listed for Delta-sarcoglycan-related limb-girdle muscular dystrophy R6.

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No actively recruiting trials found for Delta-sarcoglycan-related limb-girdle muscular dystrophy R6 at this time.

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Search ClinicalTrials.gov ↗Join the Delta-sarcoglycan-related limb-girdle muscular dystrophy R6 community →

No specialists are currently listed for Delta-sarcoglycan-related limb-girdle muscular dystrophy R6.

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 Delta-sarcoglycan-related limb-girdle muscular dystrophy R6.

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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 mutation in the SGCD gene does my family member have, and what does that mean for how the disease might progress?,How often should we have heart and lung check-ups, and what warning signs should prompt us to seek care sooner?,Is physical therapy recommended, and are there any exercises we should avoid?,Are there any clinical trials or research studies we might be eligible for?,Should other family members be tested for this condition?,What mobility aids or home modifications should we start thinking about now?,Are there patient registries or support groups you recommend we connect with?

Common questions about Delta-sarcoglycan-related limb-girdle muscular dystrophy R6

What is Delta-sarcoglycan-related limb-girdle muscular dystrophy R6?

Delta-sarcoglycan-related limb-girdle muscular dystrophy R6 (also called LGMD R6 or LGMD2F) is a rare inherited muscle disease caused by changes in the SGCD gene. This gene provides instructions for making a protein called delta-sarcoglycan, which is an important building block of a complex that protects muscle fibers during movement. When this protein is missing or not working properly, muscle fibers become fragile and break down over time. The disease mainly affects the muscles around the hips, thighs, and shoulders — the so-called 'limb-girdle' muscles. People with LGMD R6 typically notice

How is Delta-sarcoglycan-related limb-girdle muscular dystrophy R6 inherited?

Delta-sarcoglycan-related limb-girdle muscular dystrophy R6 follows a autosomal recessive inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.

At what age does Delta-sarcoglycan-related limb-girdle muscular dystrophy R6 typically begin?

Typical onset of Delta-sarcoglycan-related limb-girdle muscular dystrophy R6 is childhood. Age of onset can vary across affected individuals.