Spastic paraplegia-optic atrophy-neuropathy syndrome

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ORPHA:320406OMIM:609541G11.4
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1Specialists8Treatment centers

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Overview

Spastic paraplegia-optic atrophy-neuropathy syndrome (also known as SPOAN syndrome) is a very rare inherited neurological condition that affects the nervous system in multiple ways. The disease causes progressive stiffness and weakness in the legs (spastic paraplegia), gradual loss of vision due to damage to the optic nerves (optic atrophy), and damage to the peripheral nerves that connect the brain and spinal cord to the rest of the body (peripheral neuropathy). Symptoms typically begin in early childhood and worsen over time. People with this condition usually develop difficulty walking due to increasing leg stiffness and weakness. Vision problems may start early and can progress to significant visual impairment. The peripheral neuropathy can cause numbness, tingling, or pain in the hands and feet, as well as muscle wasting in the limbs. Some individuals may also experience problems with bladder control and other neurological symptoms. There is currently no cure for this syndrome. Treatment focuses on managing symptoms and maintaining quality of life. Physical therapy, occupational therapy, and assistive devices can help with mobility. Low-vision aids and rehabilitation services may support those with visual impairment. Medications may be used to manage spasticity and neuropathic pain. Research is ongoing to better understand the disease and develop more targeted treatments.

Also known as:

Key symptoms:

Progressive leg stiffness and difficulty walkingVision loss due to optic nerve damageNumbness or tingling in hands and feetMuscle weakness in the legsMuscle wasting in the limbsDifficulty with balance and coordinationExaggerated reflexes in the legsJoint contractures (tightening of joints)Bladder control problemsFoot deformities such as high arches or curled toesStartling response to sounds (exaggerated startle reflex)Difficulty with fine motor tasksBack pain or spinal curvature

Clinical phenotype terms (20)— hover any for plain English
Exaggerated startle responseHP:0002267Hyporeflexia of lower limbsHP:0002600Decreased number of peripheral myelinated nerve fibersHP:0003380Proximal hyperreflexiaHP:0007054
Inheritance

Autosomal recessive

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

Age of Onset

Infantile

Begins in infancy, roughly 1 month to 2 years old

Orphanet ↗OMIM ↗NORD ↗

Treatments

No FDA-approved treatments are currently listed for Spastic paraplegia-optic atrophy-neuropathy syndrome.

View clinical trials →

No actively recruiting trials found for Spastic paraplegia-optic atrophy-neuropathy syndrome at this time.

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Search ClinicalTrials.gov ↗Join the Spastic paraplegia-optic atrophy-neuropathy syndrome community →

Specialists

1 foundView all specialists →
LM
Laurent Servais, MD
Specialist
PI on 6 active trials1 Spastic paraplegia-optic atrophy-neuropathy syndrome publication

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 Spastic paraplegia-optic atrophy-neuropathy syndrome.

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Community

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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 rate of progression for my child's symptoms?,What therapies are most important to start right away?,Are there any clinical trials or research studies we could participate in?,How often should we have eye exams and neurological evaluations?,What medications can help with spasticity and nerve pain, and what are the side effects?,Should other family members be tested for the KLC2 gene mutation?,What resources are available for school accommodations and assistive technology?

Common questions about Spastic paraplegia-optic atrophy-neuropathy syndrome

What is Spastic paraplegia-optic atrophy-neuropathy syndrome?

Spastic paraplegia-optic atrophy-neuropathy syndrome (also known as SPOAN syndrome) is a very rare inherited neurological condition that affects the nervous system in multiple ways. The disease causes progressive stiffness and weakness in the legs (spastic paraplegia), gradual loss of vision due to damage to the optic nerves (optic atrophy), and damage to the peripheral nerves that connect the brain and spinal cord to the rest of the body (peripheral neuropathy). Symptoms typically begin in early childhood and worsen over time. People with this condition usually develop difficulty walking due

How is Spastic paraplegia-optic atrophy-neuropathy syndrome inherited?

Spastic paraplegia-optic atrophy-neuropathy syndrome follows a autosomal recessive inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.

At what age does Spastic paraplegia-optic atrophy-neuropathy syndrome typically begin?

Typical onset of Spastic paraplegia-optic atrophy-neuropathy syndrome is infantile. Age of onset can vary across affected individuals.

Which specialists treat Spastic paraplegia-optic atrophy-neuropathy syndrome?

1 specialists and care centers treating Spastic paraplegia-optic atrophy-neuropathy syndrome are listed on UniteRare, sourced from ClinicalTrials.gov principal investigators, published research, and the NPPES NPI registry.