Hypotonia with lactic acidemia and hyperammonemia

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ORPHA:137908OMIM:611719E88.8
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Overview

Hypotonia with lactic acidemia and hyperammonemia is a very rare metabolic condition that typically appears in newborns or young infants. The name describes its three main features: hypotonia (low muscle tone or floppiness), lactic acidemia (a buildup of lactic acid in the blood), and hyperammonemia (too much ammonia in the blood). These chemical imbalances suggest that the body's cells are not properly converting food into energy, which is a process that takes place mainly in tiny structures inside cells called mitochondria. Babies with this condition often appear floppy and weak at birth or shortly after. They may have difficulty feeding, poor weight gain, and developmental delays. The buildup of lactic acid and ammonia can be toxic to the brain and other organs if not managed. In some cases, the condition can lead to seizures, breathing difficulties, and life-threatening metabolic crises. The exact genetic cause of this condition is not fully established in all cases, and it may represent a group of related disorders rather than a single disease. Treatment is mainly supportive and focuses on managing the metabolic abnormalities, preventing crises, and supporting development. Because this condition is so rare, there is limited research on long-term outcomes, and management often requires a team of specialists working together.

Also known as:

Key symptoms:

Low muscle tone (floppiness)Poor feeding or difficulty suckingFailure to thrive or poor weight gainDevelopmental delaysSeizuresBreathing difficultiesLethargy or excessive sleepinessVomitingHigh levels of lactic acid in the bloodHigh ammonia levels in the bloodMuscle weaknessPoor reflexesIrritability

Inheritance

Variable

Can be inherited in different ways depending on the underlying gene

Age of Onset

Neonatal

Begins at or shortly after birth (first 4 weeks)

Orphanet ↗OMIM ↗NORD ↗

Treatments

No FDA-approved treatments are currently listed for Hypotonia with lactic acidemia and hyperammonemia.

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No actively recruiting trials found for Hypotonia with lactic acidemia and hyperammonemia at this time.

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No specialists are currently listed for Hypotonia with lactic acidemia and hyperammonemia.

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 Hypotonia with lactic acidemia and hyperammonemia.

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Community

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Latest news about Hypotonia with lactic acidemia and hyperammonemia

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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 is the specific underlying cause of my child's condition, and has genetic testing identified a mutation?,What are the warning signs of a metabolic crisis, and what should I do if one occurs?,What dietary changes or restrictions does my child need?,What therapies and support services are recommended for my child's development?,Are there any clinical trials or new treatments being studied for this condition?,How often should my child have blood tests to monitor lactic acid and ammonia levels?,What is the long-term outlook for my child, and how can we best support their quality of life?

Common questions about Hypotonia with lactic acidemia and hyperammonemia

What is Hypotonia with lactic acidemia and hyperammonemia?

Hypotonia with lactic acidemia and hyperammonemia is a very rare metabolic condition that typically appears in newborns or young infants. The name describes its three main features: hypotonia (low muscle tone or floppiness), lactic acidemia (a buildup of lactic acid in the blood), and hyperammonemia (too much ammonia in the blood). These chemical imbalances suggest that the body's cells are not properly converting food into energy, which is a process that takes place mainly in tiny structures inside cells called mitochondria. Babies with this condition often appear floppy and weak at birth or

At what age does Hypotonia with lactic acidemia and hyperammonemia typically begin?

Typical onset of Hypotonia with lactic acidemia and hyperammonemia is neonatal. Age of onset can vary across affected individuals.