Disorder of beta and omega amino acid metabolism

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ORPHA:308407
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Overview

Disorder of beta and omega amino acid metabolism is a group of rare inherited metabolic conditions that affect how the body breaks down certain types of amino acids. Amino acids are the building blocks of proteins, and the body normally processes them through specific chemical pathways. In these disorders, the enzymes responsible for breaking down beta amino acids (such as beta-alanine) and omega amino acids are missing or not working properly. This leads to a buildup of these substances or their byproducts in the blood and urine, which can be harmful to the body over time. Symptoms can vary widely depending on the specific enzyme affected, but may include developmental delays, seizures, muscle weakness, and problems with growth. Some individuals may have neurological symptoms such as intellectual disability or movement difficulties. The severity ranges from mild to severe. Treatment is generally supportive and focused on managing symptoms. Dietary modifications may help reduce the buildup of harmful substances. Some patients benefit from vitamin supplements or cofactor therapy. Because these are very rare conditions, there is limited research on specific treatments, and management is often individualized based on each patient's symptoms and biochemical findings. Early diagnosis through newborn screening or metabolic testing can help guide treatment and improve outcomes.

Key symptoms:

Developmental delaySeizuresMuscle weaknessIntellectual disabilityPoor growth or failure to thriveMovement problems or poor coordinationUnusual levels of amino acids in urineFatigue or low energyFeeding difficulties in infancyBehavioral changesSpeech delaysLow muscle tone (floppiness)

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 Disorder of beta and omega amino acid metabolism.

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No actively recruiting trials found for Disorder of beta and omega amino acid metabolism at this time.

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No specialists are currently listed for Disorder of beta and omega amino acid metabolism.

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 Disorder of beta and omega amino acid metabolism.

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Community

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Latest news about Disorder of beta and omega amino acid metabolism

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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.What specific type of beta or omega amino acid disorder does my child have?,Are there dietary changes that could help manage this condition?,What signs of a metabolic crisis should I watch for, and what should I do if one occurs?,How often should we have follow-up metabolic testing?,Are there any clinical trials or new treatments being studied for this condition?,What therapies (physical, occupational, speech) would benefit my child?,Should other family members be tested for this condition?

Common questions about Disorder of beta and omega amino acid metabolism

What is Disorder of beta and omega amino acid metabolism?

Disorder of beta and omega amino acid metabolism is a group of rare inherited metabolic conditions that affect how the body breaks down certain types of amino acids. Amino acids are the building blocks of proteins, and the body normally processes them through specific chemical pathways. In these disorders, the enzymes responsible for breaking down beta amino acids (such as beta-alanine) and omega amino acids are missing or not working properly. This leads to a buildup of these substances or their byproducts in the blood and urine, which can be harmful to the body over time. Symptoms can vary

How is Disorder of beta and omega amino acid metabolism inherited?

Disorder of beta and omega amino acid metabolism follows a autosomal recessive inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.