Autosomal recessive spastic paraplegia type 55

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ORPHA:320375OMIM:615035G11.4
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Overview

Autosomal recessive spastic paraplegia type 55 (SPG55) is a very rare inherited neurological condition that primarily affects the nervous system, leading to progressive stiffness and weakness in the legs. It is also known as hereditary spastic paraplegia type 55 or SPG55. The disease is caused by mutations in the C12orf65 gene, which plays an important role in how mitochondria (the energy-producing parts of cells) make proteins. When this gene does not work properly, nerve cells and other tissues that need a lot of energy are affected. People with SPG55 typically develop symptoms in childhood, though the age of onset can vary. The hallmark feature is progressive spasticity (stiffness) in the legs, which makes walking increasingly difficult over time. Many affected individuals also experience additional neurological problems, including optic atrophy (damage to the nerve connecting the eye to the brain, leading to vision loss), intellectual disability, and peripheral neuropathy (damage to nerves outside the brain and spinal cord). Some patients may also have short stature and other features. There is currently no cure for SPG55. Treatment focuses on managing symptoms and improving quality of life. Physical therapy, occupational therapy, and assistive devices can help maintain mobility and independence. Medications may be used to reduce muscle stiffness. Because the disease affects multiple body systems, a team of specialists is usually needed to provide comprehensive care.

Also known as:

Key symptoms:

Progressive leg stiffness (spasticity)Difficulty walkingWeakness in the legsVision loss due to optic nerve damage (optic atrophy)Intellectual disability or learning difficultiesNumbness or tingling in hands and feet (peripheral neuropathy)Muscle wasting in the legsShort statureDifficulty with balance and coordinationSpeech difficultiesAbnormal eye movementsFatigue

Clinical phenotype terms (27)— hover any for plain English
Optic neuropathyHP:0001138Onion bulb formationHP:0003383Decreased sensory nerve conduction velocityHP:0003448Focal white matter lesionsHP:0007042Tibialis muscle weaknessHP:0008963
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 Autosomal recessive spastic paraplegia type 55.

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No actively recruiting trials found for Autosomal recessive spastic paraplegia type 55 at this time.

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No specialists are currently listed for Autosomal recessive spastic paraplegia type 55.

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 Autosomal recessive spastic paraplegia type 55.

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Community

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Caregiver Resources

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Questions for your doctor

Bring these to your next appointment

  • Q1.What is the expected progression of symptoms for my child or family member?,How often should we schedule physical therapy and eye exams?,Are there any medications that can help with the leg stiffness?,What educational or developmental support services should we look into?,Are there any clinical trials or research studies we could participate in?,Should other family members be tested for carrier status?,What signs should prompt us to seek urgent medical attention?

Common questions about Autosomal recessive spastic paraplegia type 55

What is Autosomal recessive spastic paraplegia type 55?

Autosomal recessive spastic paraplegia type 55 (SPG55) is a very rare inherited neurological condition that primarily affects the nervous system, leading to progressive stiffness and weakness in the legs. It is also known as hereditary spastic paraplegia type 55 or SPG55. The disease is caused by mutations in the C12orf65 gene, which plays an important role in how mitochondria (the energy-producing parts of cells) make proteins. When this gene does not work properly, nerve cells and other tissues that need a lot of energy are affected. People with SPG55 typically develop symptoms in childhood

How is Autosomal recessive spastic paraplegia type 55 inherited?

Autosomal recessive spastic paraplegia type 55 follows a autosomal recessive inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.

At what age does Autosomal recessive spastic paraplegia type 55 typically begin?

Typical onset of Autosomal recessive spastic paraplegia type 55 is childhood. Age of onset can vary across affected individuals.