Proximal myopathy with extrapyramidal signs

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ORPHA:401768OMIM:615673G71.3
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Overview

Proximal myopathy with extrapyramidal signs (PMES) is an extremely rare genetic condition that affects both the muscles and the movement control centers of the brain. The disease causes weakness in the muscles closest to the center of the body, such as the shoulders, upper arms, hips, and thighs (called proximal muscles). In addition, people with this condition develop extrapyramidal signs, which are movement problems that can include stiffness, tremors, slow movements, and difficulty coordinating voluntary actions — similar to what is seen in Parkinson's disease or other movement disorders. Symptoms typically begin in adulthood and tend to get worse over time. People may first notice difficulty climbing stairs, rising from a chair, or lifting their arms above their heads. As the disease progresses, the movement abnormalities become more noticeable, and daily activities can become increasingly challenging. Some patients may also experience difficulty swallowing or speaking. Because this condition is so rare, treatment options are currently limited to managing symptoms. Physical therapy, occupational therapy, and medications that address movement problems may help improve quality of life. There is no cure at this time, and research into this condition is still in its early stages. Genetic counseling is recommended for affected families to understand the inheritance pattern and risks to other family members.

Key symptoms:

Weakness in the shoulders and upper armsWeakness in the hips and thighsMuscle stiffness or rigidityTremorSlow movementsDifficulty walkingTrouble climbing stairsDifficulty rising from a seated positionProblems with balance and coordinationDifficulty swallowingSpeech difficultiesMuscle wasting over timeInvoluntary movements

Clinical phenotype terms (25)— hover any for plain English
Orofacial dyskinesiaHP:0002310Resting tremorHP:0002322Centrally nucleated skeletal muscle fibersHP:0003687Progressive extrapyramidal movement disorderHP:0007153Progressive extrapyramidal muscular rigidityHP:0007158Abnormal basal ganglia MRI signal intensityHP:0012751Central core regions in muscle fibersHP:0030230
Inheritance

Autosomal recessive

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

Age of Onset

Adult

Begins in adulthood (age 18 or older)

Orphanet ↗OMIM ↗NORD ↗

Treatments

No FDA-approved treatments are currently listed for Proximal myopathy with extrapyramidal signs.

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No actively recruiting trials found for Proximal myopathy with extrapyramidal signs at this time.

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No specialists are currently listed for Proximal myopathy with extrapyramidal signs.

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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

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Community

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Caregiver Resources

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Questions for your doctor

Bring these to your next appointment

  • Q1.What specific genetic testing should I have to confirm this diagnosis?,How quickly is this condition likely to progress in my case?,Are there any medications that might help with my movement symptoms?,What type of physical therapy program would be most beneficial for me?,Should my family members be tested for this condition?,Are there any clinical trials or research studies I could participate in?,What support services are available to help me maintain my independence?

Common questions about Proximal myopathy with extrapyramidal signs

What is Proximal myopathy with extrapyramidal signs?

Proximal myopathy with extrapyramidal signs (PMES) is an extremely rare genetic condition that affects both the muscles and the movement control centers of the brain. The disease causes weakness in the muscles closest to the center of the body, such as the shoulders, upper arms, hips, and thighs (called proximal muscles). In addition, people with this condition develop extrapyramidal signs, which are movement problems that can include stiffness, tremors, slow movements, and difficulty coordinating voluntary actions — similar to what is seen in Parkinson's disease or other movement disorders.

How is Proximal myopathy with extrapyramidal signs inherited?

Proximal myopathy with extrapyramidal signs follows a autosomal recessive inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.

At what age does Proximal myopathy with extrapyramidal signs typically begin?

Typical onset of Proximal myopathy with extrapyramidal signs is adult. Age of onset can vary across affected individuals.