OBSOLETE: LIMS2-related myopathy

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ORPHA:466801
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Overview

LIMS2-related myopathy is an extremely rare inherited muscle disease caused by changes (mutations) in the LIMS2 gene. The term 'OBSOLETE' in the disease name means that this entry has been retired or reclassified in medical databases, but the condition itself is still recognized. The LIMS2 gene provides instructions for making a protein called PINCH2, which plays an important role in connecting muscle cells to the surrounding tissue and helping muscles function properly. When this gene does not work correctly, the muscles become weak and may waste away over time. Patients with this condition typically experience progressive muscle weakness, which can affect the ability to walk, climb stairs, and perform everyday tasks. The severity can vary, but the disease generally leads to increasing difficulty with movement. Some patients may also develop problems with their heart muscle or breathing muscles over time. Because this is such a rare condition, there is currently no specific cure or targeted treatment. Management focuses on supportive care, including physical therapy to maintain muscle strength and flexibility, respiratory support if breathing muscles are affected, and regular monitoring by a team of specialists. Research into this condition is still in its early stages, and understanding of the full clinical picture continues to evolve.

Key symptoms:

Progressive muscle weaknessDifficulty walking or climbing stairsMuscle wastingFatigue during physical activityDifficulty lifting arms above the headTrouble getting up from a seated or lying positionPossible breathing difficultiesPossible heart muscle involvementJoint stiffness or contracturesDelayed motor milestones in children

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 ↗NORD ↗

Treatments

No FDA-approved treatments are currently listed for OBSOLETE: LIMS2-related myopathy.

View clinical trials →

No actively recruiting trials found for OBSOLETE: LIMS2-related myopathy at this time.

New trials open frequently. Follow this disease to get notified.

Search ClinicalTrials.gov ↗Join the OBSOLETE: LIMS2-related myopathy community →

No specialists are currently listed for OBSOLETE: LIMS2-related myopathy.

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 OBSOLETE: LIMS2-related myopathy.

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Community

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Latest news about OBSOLETE: LIMS2-related myopathy

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Caregiver Resources

NORD Caregiver Resources

Support, advocacy, and financial assistance for caregivers of rare disease patients.

Mental Health Support

Rare disease caregiving can be isolating. Connect with counseling and peer support.

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 the muscle involvement at this point, and what is the expected rate of progression?,Should my heart and lungs be monitored regularly, and how often?,What types of physical therapy or exercise are safe and beneficial?,Are there any clinical trials or research studies I could participate in?,Should other family members be tested for the LIMS2 gene mutation?,What assistive devices or home modifications might help as the disease progresses?,Are there any medications I should avoid that could worsen muscle weakness?

Common questions about OBSOLETE: LIMS2-related myopathy

What is OBSOLETE: LIMS2-related myopathy?

LIMS2-related myopathy is an extremely rare inherited muscle disease caused by changes (mutations) in the LIMS2 gene. The term 'OBSOLETE' in the disease name means that this entry has been retired or reclassified in medical databases, but the condition itself is still recognized. The LIMS2 gene provides instructions for making a protein called PINCH2, which plays an important role in connecting muscle cells to the surrounding tissue and helping muscles function properly. When this gene does not work correctly, the muscles become weak and may waste away over time. Patients with this condition

How is OBSOLETE: LIMS2-related myopathy inherited?

OBSOLETE: LIMS2-related myopathy follows a autosomal recessive inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.