Lethal neonatal spasticity-epileptic encephalopathy syndrome

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ORPHA:435845OMIM:618056G40.4
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Overview

Lethal neonatal spasticity-epileptic encephalopathy syndrome is an extremely rare and severe brain disorder that appears at birth or within the first days of life. It is characterized by a combination of severe muscle stiffness (spasticity), uncontrollable seizures (epileptic encephalopathy), and profound brain dysfunction. Affected newborns typically show very poor muscle tone or extreme stiffness, frequent and difficult-to-control seizures, and little to no normal brain activity. The condition is caused by genetic changes that disrupt normal brain development and function, particularly affecting how nerve cells communicate with each other. Because this condition is described as lethal, affected infants generally have a very poor prognosis, with most not surviving beyond the newborn period or early infancy. The seizures in this condition tend to be resistant to standard anti-seizure medications, making treatment extremely challenging. Care is primarily supportive, focusing on comfort, seizure management, and respiratory support. There are currently no curative treatments available. Families affected by this condition benefit greatly from genetic counseling and palliative care support to help navigate this devastating diagnosis.

Also known as:

Key symptoms:

Severe muscle stiffness (spasticity)Frequent and hard-to-control seizuresSevere brain dysfunction from birthPoor or absent voluntary movementsDifficulty breathing or need for breathing supportFeeding difficultiesLack of normal newborn reflexesLittle or no response to surroundingsAbnormal brain wave patterns on EEGFailure to reach any developmental milestonesLow muscle tone alternating with stiffnessIrritability or excessive crying

Inheritance

Autosomal recessive

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

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 Lethal neonatal spasticity-epileptic encephalopathy syndrome.

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No actively recruiting trials found for Lethal neonatal spasticity-epileptic encephalopathy syndrome at this time.

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No specialists are currently listed for Lethal neonatal spasticity-epileptic encephalopathy syndrome.

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 Lethal neonatal spasticity-epileptic encephalopathy syndrome.

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Community

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Caregiver Resources

NORD Caregiver Resources

Support, advocacy, and financial assistance for caregivers of rare disease patients.

Mental Health Support

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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 genetic change is causing my baby's condition?,Are there any treatments that might help control the seizures?,What is the expected course of this condition for my baby?,Can we involve a palliative care team to help with comfort and support?,What does this diagnosis mean for future pregnancies, and should we pursue genetic counseling?,Are there any clinical trials or research studies we could participate in?,What support services are available for our family during this time?

Common questions about Lethal neonatal spasticity-epileptic encephalopathy syndrome

What is Lethal neonatal spasticity-epileptic encephalopathy syndrome?

Lethal neonatal spasticity-epileptic encephalopathy syndrome is an extremely rare and severe brain disorder that appears at birth or within the first days of life. It is characterized by a combination of severe muscle stiffness (spasticity), uncontrollable seizures (epileptic encephalopathy), and profound brain dysfunction. Affected newborns typically show very poor muscle tone or extreme stiffness, frequent and difficult-to-control seizures, and little to no normal brain activity. The condition is caused by genetic changes that disrupt normal brain development and function, particularly affec

How is Lethal neonatal spasticity-epileptic encephalopathy syndrome inherited?

Lethal neonatal spasticity-epileptic encephalopathy syndrome follows a autosomal recessive inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.

At what age does Lethal neonatal spasticity-epileptic encephalopathy syndrome typically begin?

Typical onset of Lethal neonatal spasticity-epileptic encephalopathy syndrome is neonatal. Age of onset can vary across affected individuals.