X-linked complicated spastic paraplegia type 1

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Overview

X-linked complicated spastic paraplegia type 1 (also known as SPG1 or spastic paraplegia 1) is a rare inherited neurological condition that primarily affects males. It is caused by mutations in the L1CAM gene on the X chromosome. This gene plays a critical role in the development of the nervous system, particularly in how nerve cells grow, connect, and communicate with each other. The hallmark feature of this condition is progressive stiffness and weakness in the legs (spastic paraplegia), but because it is a "complicated" form, it comes with additional neurological problems. These can include intellectual disability, difficulty speaking, hydrocephalus (excess fluid in the brain), and problems with the structure of the brain such as absence or thinning of the corpus callosum (the bridge connecting the two halves of the brain). Some affected individuals may also have adducted (inward-turning) thumbs, which is a distinctive feature associated with L1CAM mutations. Symptoms typically begin in infancy or early childhood. The severity can vary widely, even among family members carrying the same mutation. There is currently no cure for SPG1. Treatment focuses on managing symptoms and maximizing quality of life through physical therapy, medications to reduce muscle stiffness, surgical interventions for hydrocephalus if needed, and educational support for intellectual challenges. A team of specialists is usually needed to provide comprehensive care.

Also known as:

Key symptoms:

Progressive leg stiffness and weaknessDifficulty walking or inability to walkIntellectual disabilityHydrocephalus (excess fluid in the brain)Adducted (inward-turning) thumbsSpeech and language delaysAbsent or thin corpus callosum in the brainIncreased muscle tone in the legs (spasticity)Difficulty with fine motor skillsSeizures in some casesUrinary problemsDelayed developmental milestonesPoor coordination and balance

Clinical phenotype terms (9)— hover any for plain English
Inheritance

X-linked recessive

Carried on the X chromosome; typically affects males more than females

Age of Onset

Infantile

Begins in infancy, roughly 1 month to 2 years old

Orphanet ↗NORD ↗

Treatments

No FDA-approved treatments are currently listed for X-linked complicated spastic paraplegia type 1.

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No actively recruiting trials found for X-linked complicated spastic paraplegia type 1 at this time.

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No specialists are currently listed for X-linked complicated spastic paraplegia type 1.

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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

No travel grants are currently matched to X-linked complicated spastic paraplegia type 1.

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Questions for your doctor

Bring these to your next appointment

  • Q1.What is the specific L1CAM mutation in our family, and what does it mean for severity?,Does my child have hydrocephalus, and will a shunt be needed?,What therapies should we start right away to support development?,Are there clinical trials or research studies we should know about?,What are the signs of a medical emergency I should watch for?,Should other family members be tested for carrier status?,What is the long-term outlook for mobility and independence in my child's case?

Common questions about X-linked complicated spastic paraplegia type 1

What is X-linked complicated spastic paraplegia type 1?

X-linked complicated spastic paraplegia type 1 (also known as SPG1 or spastic paraplegia 1) is a rare inherited neurological condition that primarily affects males. It is caused by mutations in the L1CAM gene on the X chromosome. This gene plays a critical role in the development of the nervous system, particularly in how nerve cells grow, connect, and communicate with each other. The hallmark feature of this condition is progressive stiffness and weakness in the legs (spastic paraplegia), but because it is a "complicated" form, it comes with additional neurological problems. These can includ

How is X-linked complicated spastic paraplegia type 1 inherited?

X-linked complicated spastic paraplegia type 1 follows a x-linked recessive inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.

At what age does X-linked complicated spastic paraplegia type 1 typically begin?

Typical onset of X-linked complicated spastic paraplegia type 1 is infantile. Age of onset can vary across affected individuals.