Atypical glycine encephalopathy

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ORPHA:289863OMIM:617301E72.5
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Overview

Atypical glycine encephalopathy, also known as atypical nonketotic hyperglycinemia (atypical NKH), is a rare inherited metabolic disorder that affects how the body breaks down the amino acid glycine. Unlike the more common classic form of glycine encephalopathy, the atypical form may present later in life or with milder or unusual symptoms. In this condition, glycine builds up to high levels in the body, especially in the brain and spinal fluid, which can damage the nervous system. Symptoms can vary widely but often include developmental delay, intellectual disability, seizures, and problems with muscle tone. Some individuals may have movement disorders such as chorea (involuntary jerky movements) or spasticity. Because the presentation is less severe or different from the classic neonatal form, diagnosis can be delayed. There is currently no cure for atypical glycine encephalopathy. Treatment focuses on managing symptoms and trying to lower glycine levels in the body. Medications such as sodium benzoate are used to reduce glycine levels, and dextromethorphan may be used to block the effects of excess glycine at brain receptors. Anti-seizure medications are often needed to control epilepsy. Early diagnosis and treatment may improve outcomes, but the disease course varies significantly from person to person.

Also known as:

Key symptoms:

Seizures or epilepsyDevelopmental delayIntellectual disabilityLow muscle tone (floppiness)Increased muscle stiffness (spasticity)Involuntary jerky movements (chorea)Difficulty with coordination and balanceSpeech and language delaysBehavioral problemsDifficulty feedingBreathing problems in some casesLearning difficultiesAttention problems

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 Atypical glycine encephalopathy.

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No actively recruiting trials found for Atypical glycine encephalopathy at this time.

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No specialists are currently listed for Atypical glycine encephalopathy.

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 Atypical glycine encephalopathy.

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Community

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Caregiver Resources

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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 type of atypical glycine encephalopathy does my child have, and which gene is affected?,What is the expected course of the disease based on the specific genetic variant found?,What medications should we start, and what side effects should we watch for?,Are there any medications or foods we need to strictly avoid?,How often should glycine levels be monitored, and what are the target levels?,What therapies (physical, occupational, speech) are recommended and how often?,Are there any clinical trials or new treatments being studied that we should know about?

Common questions about Atypical glycine encephalopathy

What is Atypical glycine encephalopathy?

Atypical glycine encephalopathy, also known as atypical nonketotic hyperglycinemia (atypical NKH), is a rare inherited metabolic disorder that affects how the body breaks down the amino acid glycine. Unlike the more common classic form of glycine encephalopathy, the atypical form may present later in life or with milder or unusual symptoms. In this condition, glycine builds up to high levels in the body, especially in the brain and spinal fluid, which can damage the nervous system. Symptoms can vary widely but often include developmental delay, intellectual disability, seizures, and problems

How is Atypical glycine encephalopathy inherited?

Atypical glycine encephalopathy follows a autosomal recessive inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.