Moderate multiminicore disease with hand involvement

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ORPHA:178145OMIM:117000G71.2
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Overview

Moderate multiminicore disease with hand involvement is a rare inherited muscle disorder that belongs to a group of conditions called multiminicore diseases (also known as multicore myopathies or multi-minicore disease). These conditions are named after the tiny areas of damage, called 'minicores,' that can be seen in muscle tissue under a microscope. In this particular form, the muscle weakness is moderate in severity and notably affects the hands in addition to other muscles in the body. People with this condition typically experience weakness in the muscles of the hands, which can make fine motor tasks like gripping, writing, or buttoning clothes more difficult. There is also more widespread muscle weakness that can affect the limbs and trunk. The degree of weakness is considered moderate, meaning that while it does limit physical abilities, many individuals retain a significant level of function and independence. Currently, there is no cure for this condition. Treatment focuses on managing symptoms and maintaining function through physical therapy, occupational therapy (especially for hand function), and regular monitoring by specialists. Supportive devices and adaptive tools may help with daily tasks. Research into the genetic causes and potential therapies for multiminicore diseases is ongoing, but specific targeted treatments are not yet available for this subtype.

Key symptoms:

Moderate muscle weakness throughout the bodyWeakness and reduced function in the handsDifficulty with fine motor tasks like writing or gripping objectsReduced muscle bulk (muscle wasting)Joint stiffness or limited range of motionDelayed motor milestones in childhoodFatigue during physical activityDifficulty with activities requiring hand strengthPossible mild scoliosis (curvature of the spine)Reduced exercise tolerance

Clinical phenotype terms (14)— hover any for plain English
Intrinsic hand muscle atrophyHP:0008954Distal upper limb muscle weaknessHP:0008959Hyporeflexia of upper limbsHP:0012391Type 1 muscle fiber predominanceHP:0003803Knee dislocationHP:0004976Recurrent patellar dislocationHP:0005001
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 Moderate multiminicore disease with hand involvement.

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No actively recruiting trials found for Moderate multiminicore disease with hand involvement at this time.

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No specialists are currently listed for Moderate multiminicore disease with hand involvement.

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 Moderate multiminicore disease with hand involvement.

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Caregiver Resources

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Questions for your doctor

Bring these to your next appointment

  • Q1.What specific genetic mutation is causing this condition in my case or my child's case?,How should we monitor for changes in muscle strength or breathing over time?,What types of physical and occupational therapy are most helpful for hand involvement?,Are there any clinical trials or emerging treatments we should know about?,What school or workplace accommodations would you recommend?,How often should we have follow-up appointments and what tests are needed?,Should other family members be tested for this genetic condition?

Common questions about Moderate multiminicore disease with hand involvement

What is Moderate multiminicore disease with hand involvement?

Moderate multiminicore disease with hand involvement is a rare inherited muscle disorder that belongs to a group of conditions called multiminicore diseases (also known as multicore myopathies or multi-minicore disease). These conditions are named after the tiny areas of damage, called 'minicores,' that can be seen in muscle tissue under a microscope. In this particular form, the muscle weakness is moderate in severity and notably affects the hands in addition to other muscles in the body. People with this condition typically experience weakness in the muscles of the hands, which can make fin

How is Moderate multiminicore disease with hand involvement inherited?

Moderate multiminicore disease with hand involvement follows a autosomal recessive inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.

At what age does Moderate multiminicore disease with hand involvement typically begin?

Typical onset of Moderate multiminicore disease with hand involvement is childhood. Age of onset can vary across affected individuals.