Combined oxidative phosphorylation defect type 23

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ORPHA:444013OMIM:616198E88.8
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Overview

Combined oxidative phosphorylation defect type 23 (also written as COXPD23) is a rare inherited disorder that affects the way cells produce energy. Every cell in the body relies on tiny structures called mitochondria to convert food into usable energy. In COXPD23, a faulty gene disrupts this energy-making process, specifically affecting a system called the mitochondrial ribosome, which is needed to build the proteins that power the cell's energy factories. When this system does not work properly, cells — especially those in the brain, muscles, and liver — do not get enough energy to function normally. This condition is caused by changes (mutations) in the GTPBP5 gene. It typically appears in infancy or early childhood and can cause a wide range of serious problems. Children with COXPD23 may show signs of brain dysfunction, muscle weakness, poor growth, and problems with multiple organs. The severity can vary from child to child. Because this is a mitochondrial disease, it tends to affect organs that need the most energy. There is currently no cure for COXPD23. Treatment focuses on managing symptoms, supporting nutrition, and protecting organ function. A team of specialists is usually needed to care for affected individuals. Research into mitochondrial diseases is ongoing, and some supportive therapies — such as certain vitamins and supplements — may be used, though their benefit in this specific condition is not yet fully proven.

Also known as:

Key symptoms:

muscle weakness or poor muscle tone (hypotonia)delayed development or regression of milestonesintellectual disability or learning difficultiespoor feeding and failure to thriveliver problems (hepatopathy)elevated lactic acid in the blood (lactic acidosis)breathing difficultiesseizuresabnormal brain structure seen on MRI (brain lesions)poor growth and low weightfatigue and low energymovement problems or coordination difficulties

Clinical phenotype terms (27)— hover any for plain English
Right ventricular hypertrophyHP:0001667Wolff-Parkinson-White syndromeHP:0001716LeukodystrophyHP:0002415Easy fatigabilityHP:0003388Decreased activity of mitochondrial complex IVHP:0008347
Inheritance

Autosomal recessive

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

Age of Onset

Infantile

Begins in infancy, roughly 1 month to 2 years old

Orphanet ↗OMIM ↗NORD ↗

Treatments

No FDA-approved treatments are currently listed for Combined oxidative phosphorylation defect type 23.

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No actively recruiting trials found for Combined oxidative phosphorylation defect type 23 at this time.

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No specialists are currently listed for Combined oxidative phosphorylation defect type 23.

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 Combined oxidative phosphorylation defect type 23.

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Community

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Caregiver Resources

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Social Security Disability

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Questions for your doctor

Bring these to your next appointment

  • Q1.Which organs are currently affected in my child, and how will we monitor them over time?,Should my child be taking mitochondrial supplements, and if so, which ones and at what doses?,What are the warning signs of a metabolic crisis, and what should I do if one happens?,Are there any clinical trials or research studies that my child might be eligible for?,What therapies (physical, occupational, speech) would benefit my child most right now?,Should other family members be tested for this condition?,What is the long-term care plan, and which specialists should we see regularly?

Common questions about Combined oxidative phosphorylation defect type 23

What is Combined oxidative phosphorylation defect type 23?

Combined oxidative phosphorylation defect type 23 (also written as COXPD23) is a rare inherited disorder that affects the way cells produce energy. Every cell in the body relies on tiny structures called mitochondria to convert food into usable energy. In COXPD23, a faulty gene disrupts this energy-making process, specifically affecting a system called the mitochondrial ribosome, which is needed to build the proteins that power the cell's energy factories. When this system does not work properly, cells — especially those in the brain, muscles, and liver — do not get enough energy to function n

How is Combined oxidative phosphorylation defect type 23 inherited?

Combined oxidative phosphorylation defect type 23 follows a autosomal recessive inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.

At what age does Combined oxidative phosphorylation defect type 23 typically begin?

Typical onset of Combined oxidative phosphorylation defect type 23 is infantile. Age of onset can vary across affected individuals.