Metabolic myopathy due to lactate transporter defect

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ORPHA:171690OMIM:245340G72.8
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Overview

Metabolic myopathy due to lactate transporter defect is an extremely rare inherited muscle disease caused by a problem with how the body moves lactate (a substance produced during exercise and normal metabolism) in and out of muscle cells. Lactate is an important fuel source and byproduct of energy production. When the transporter responsible for moving lactate across cell membranes does not work properly, lactate builds up in the muscles, leading to muscle problems. People with this condition typically experience exercise intolerance, meaning they become unusually tired or develop muscle pain, cramping, and weakness during physical activity. After exercise, blood lactate levels may rise much higher than expected. Some individuals may also notice chronic fatigue and difficulty performing everyday physical tasks. The severity can vary from person to person. Because this condition is so rare, treatment options are limited and mainly focus on managing symptoms. Patients are generally advised to avoid intense physical exertion, pace their activities, and work closely with specialists in metabolic diseases and neuromuscular disorders. There is currently no cure or specific approved therapy for this condition, and management is supportive in nature.

Also known as:

Key symptoms:

Exercise intoleranceMuscle pain during or after physical activityMuscle crampsMuscle weaknessChronic fatigueElevated blood lactate levels after exerciseDifficulty with sustained physical effortMuscle stiffnessShortness of breath during exertion

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 ↗OMIM ↗NORD ↗

Treatments

No FDA-approved treatments are currently listed for Metabolic myopathy due to lactate transporter defect.

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No actively recruiting trials found for Metabolic myopathy due to lactate transporter defect at this time.

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No specialists are currently listed for Metabolic myopathy due to lactate transporter defect.

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 Metabolic myopathy due to lactate transporter defect.

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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

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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 level of physical activity is safe for me or my child?,How can I recognize the early signs of muscle breakdown (rhabdomyolysis)?,Are there any dietary changes that could help manage symptoms?,Should other family members be tested for this condition?,What should I do in an emergency if severe muscle symptoms occur?,Are there any clinical trials or new treatments being studied for this condition?,How often should follow-up appointments and blood tests be scheduled?

Common questions about Metabolic myopathy due to lactate transporter defect

What is Metabolic myopathy due to lactate transporter defect?

Metabolic myopathy due to lactate transporter defect is an extremely rare inherited muscle disease caused by a problem with how the body moves lactate (a substance produced during exercise and normal metabolism) in and out of muscle cells. Lactate is an important fuel source and byproduct of energy production. When the transporter responsible for moving lactate across cell membranes does not work properly, lactate builds up in the muscles, leading to muscle problems. People with this condition typically experience exercise intolerance, meaning they become unusually tired or develop muscle pai

How is Metabolic myopathy due to lactate transporter defect inherited?

Metabolic myopathy due to lactate transporter defect follows a autosomal recessive inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.