Autosomal recessive spastic paraplegia type 67

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ORPHA:401820OMIM:615802G11.4
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Overview

Autosomal recessive spastic paraplegia type 67 (SPG67) is an extremely 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 67 or SPG67. This disease belongs to a large group of disorders called hereditary spastic paraplegias (HSPs), which all share the hallmark feature of gradually increasing muscle stiffness (spasticity) in the lower limbs. SPG67 is caused by mutations in the PGAP1 gene, which plays an important role in how certain proteins are processed and attached to the surface of cells. When this gene does not work properly, nerve cells — especially the long nerve fibers that control leg movement — can become damaged over time. People with SPG67 typically develop symptoms in early childhood, including difficulty walking, stiff legs, and sometimes intellectual disability or developmental delays. Some individuals may also experience seizures or problems with speech. There is currently no cure for SPG67. Treatment focuses on managing symptoms and improving quality of life. Physical therapy, medications to reduce muscle stiffness (such as baclofen or tizanidine), and assistive devices like braces or walkers are commonly used. Ongoing research into hereditary spastic paraplegias may eventually lead to more targeted therapies, but for now, supportive care remains the standard approach.

Also known as:

Key symptoms:

Progressive leg stiffness (spasticity)Difficulty walking or abnormal gaitMuscle weakness in the legsIntellectual disabilityDelayed developmental milestonesSpeech difficultiesSeizuresIncreased muscle reflexesBalance problemsFoot deformities such as high arches or curled toesDifficulty with fine motor skillsThin or wasted leg muscles over time

Clinical phenotype terms (15)— hover any for plain English
Generalized amyotrophyHP:0003700Aplasia/Hypoplasia of the cerebellar vermisHP:0006817Abnormal myelinationHP:0012447Limb tremorHP:0200085
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 Autosomal recessive spastic paraplegia type 67.

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No specialists are currently listed for Autosomal recessive spastic paraplegia type 67.

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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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Questions for your doctor

Bring these to your next appointment

  • Q1.What is the expected progression of symptoms for my child or family member?,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 schedule follow-up visits with the neurologist?,What medications can help with muscle stiffness, and what are their side effects?,Should other family members be tested to see if they are carriers?,What school or workplace accommodations should we request?

Common questions about Autosomal recessive spastic paraplegia type 67

What is Autosomal recessive spastic paraplegia type 67?

Autosomal recessive spastic paraplegia type 67 (SPG67) is an extremely 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 67 or SPG67. This disease belongs to a large group of disorders called hereditary spastic paraplegias (HSPs), which all share the hallmark feature of gradually increasing muscle stiffness (spasticity) in the lower limbs. SPG67 is caused by mutations in the PGAP1 gene, which plays an important role in how certain proteins are proces

How is Autosomal recessive spastic paraplegia type 67 inherited?

Autosomal recessive spastic paraplegia type 67 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 67 typically begin?

Typical onset of Autosomal recessive spastic paraplegia type 67 is infantile. Age of onset can vary across affected individuals.