Childhood-onset spasticity with hyperglycinemia

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ORPHA:401866OMIM:616859E88.8
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Overview

Childhood-onset spasticity with hyperglycinemia is an extremely rare genetic condition that affects the nervous system and metabolism. In this disease, children develop spasticity, which means their muscles become unusually stiff and tight, making movement difficult. At the same time, they have elevated levels of glycine, an amino acid, in their blood and sometimes in their spinal fluid. This combination of muscle stiffness and abnormal glycine levels points to a problem in how the body processes certain chemicals needed for normal brain and nerve function. Symptoms typically begin during childhood and may include progressive difficulty with walking, stiff legs, problems with coordination, and sometimes intellectual or developmental challenges. The severity can vary from one child to another. Because this condition is so rare, the treatment landscape is limited and mainly focuses on managing symptoms. Physical therapy, medications to reduce muscle stiffness (such as baclofen or other antispasticity drugs), and supportive care are the main approaches. There is currently no cure, and research into this condition is still in its early stages. Families affected by this disease often benefit from working closely with a team of specialists including neurologists and metabolic disease experts.

Also known as:

Key symptoms:

Stiff and tight muscles, especially in the legsDifficulty walking or abnormal walking patternIncreased muscle tone (spasticity)Elevated glycine levels in the bloodProblems with balance and coordinationDelayed motor milestonesDifficulty with fine motor tasksPossible learning difficulties or intellectual disabilityExaggerated reflexesFatigue with physical activityPossible speech difficulties

Clinical phenotype terms (25)— hover any for plain English
Progressive spasticityHP:0002191Nonketotic hyperglycinemiaHP:0008288Spastic diplegiaHP:0001264LeukodystrophyHP:0002415Decreased activity of the pyruvate dehydrogenase complexHP:0002928Loss of ability to walk in early childhoodHP:0008945Spinal cord lesionHP:0100561
Inheritance

Autosomal recessive

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

Age of Onset

Childhood

Begins in childhood, roughly ages 1 to 12

Orphanet ↗OMIM ↗NORD ↗

Treatments

No FDA-approved treatments are currently listed for Childhood-onset spasticity with hyperglycinemia.

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No actively recruiting trials found for Childhood-onset spasticity with hyperglycinemia at this time.

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No specialists are currently listed for Childhood-onset spasticity with hyperglycinemia.

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

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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.What is the expected progression of my child's symptoms over time?,Are there any specific medications that could help reduce my child's muscle stiffness?,Should we pursue genetic testing, and what type of test is most appropriate?,How often should my child's glycine levels be monitored, and what do the results mean?,What therapies (physical, occupational, speech) do you recommend, and how often?,Are there any clinical trials or research studies my child might be eligible for?,What school accommodations should we request to support my child's learning and mobility?

Common questions about Childhood-onset spasticity with hyperglycinemia

What is Childhood-onset spasticity with hyperglycinemia?

Childhood-onset spasticity with hyperglycinemia is an extremely rare genetic condition that affects the nervous system and metabolism. In this disease, children develop spasticity, which means their muscles become unusually stiff and tight, making movement difficult. At the same time, they have elevated levels of glycine, an amino acid, in their blood and sometimes in their spinal fluid. This combination of muscle stiffness and abnormal glycine levels points to a problem in how the body processes certain chemicals needed for normal brain and nerve function. Symptoms typically begin during chi

How is Childhood-onset spasticity with hyperglycinemia inherited?

Childhood-onset spasticity with hyperglycinemia follows a autosomal recessive inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.

At what age does Childhood-onset spasticity with hyperglycinemia typically begin?

Typical onset of Childhood-onset spasticity with hyperglycinemia is childhood. Age of onset can vary across affected individuals.