Autosomal recessive complex spastic paraplegia due to Kennedy pathway dysfunction

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ORPHA:506353OMIM:618768G11.4
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Overview

Autosomal recessive complex spastic paraplegia due to Kennedy pathway dysfunction is a very rare inherited neurological disease. It is sometimes referred to as a hereditary spastic paraplegia (HSP) or spastic paraplegia related to the Kennedy pathway. This condition affects the nervous system, particularly the long nerve fibers that run down the spinal cord and control movement in the legs. When these nerve fibers are damaged or do not work properly, the muscles in the legs become stiff and weak over time. The Kennedy pathway is a set of chemical reactions inside cells that helps build important fats called phospholipids, which are essential for healthy nerve cell membranes. When genes involved in this pathway are faulty, nerve cells cannot maintain their structure properly, leading to the gradual breakdown of motor function. People with this condition typically experience progressive leg stiffness and weakness, difficulty walking, and problems with balance and coordination. Some individuals also develop additional neurological features beyond the legs, which is why it is called 'complex' spastic paraplegia. Currently, there is no cure for this condition. Treatment focuses on managing symptoms, maintaining mobility, and improving quality of life through physical therapy, medications to reduce muscle stiffness, and supportive care. Research into this rare disease is ongoing, and genetic diagnosis is important for families to understand inheritance risks.

Also known as:

Key symptoms:

Progressive stiffness in the legs (spasticity)Muscle weakness in the legsDifficulty walking or an abnormal walking pattern (gait problems)Problems with balance and coordinationOveractive reflexes in the legsBladder control problems (urgency or incontinence)Fatigue, especially when walkingFoot deformities such as high arches (pes cavus)Reduced sensation or numbness in the legs in some casesCognitive or intellectual difficulties in some individualsSpeech difficulties in some casesDifficulty climbing stairs or rising from a chair

Clinical phenotype terms (22)— hover any for plain English
Moderately short statureHP:0008848Nasal dysarthriaHP:0008376Progressive spasticityHP:0002191Ankle clonusHP:0011448Hyporeflective spaces on macular OCTHP:0030625Retinal pigment epithelial mottlingHP:0007814Progressive spastic paraparesisHP:0007199Tetraplegia/tetraparesisHP:0030182Demyelinating motor neuropathyHP:0007220Central retinal vessel vascular tortuosityHP:0007768
Inheritance

Autosomal recessive

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

Age of Onset

Variable

Can begin at different ages, from infancy through adulthood

Orphanet ↗OMIM ↗NORD ↗

Treatments

No FDA-approved treatments are currently listed for Autosomal recessive complex spastic paraplegia due to Kennedy pathway dysfunction.

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No actively recruiting trials found for Autosomal recessive complex spastic paraplegia due to Kennedy pathway dysfunction at this time.

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No specialists are currently listed for Autosomal recessive complex spastic paraplegia due to Kennedy pathway dysfunction.

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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 Autosomal recessive complex spastic paraplegia due to Kennedy pathway dysfunction.

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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.Which specific gene mutation has been found in my case, and what does that mean for my prognosis?,Should other family members be tested for this condition?,What physical therapy program do you recommend to help maintain my walking ability?,Are there any clinical trials or research studies I could participate in?,What medications are available to help with my leg stiffness, and what are the side effects?,How often should I have follow-up appointments and what tests will be done to monitor progression?,Are there any patient registries or specialist centers I should connect with for this rare condition?

Common questions about Autosomal recessive complex spastic paraplegia due to Kennedy pathway dysfunction

What is Autosomal recessive complex spastic paraplegia due to Kennedy pathway dysfunction?

Autosomal recessive complex spastic paraplegia due to Kennedy pathway dysfunction is a very rare inherited neurological disease. It is sometimes referred to as a hereditary spastic paraplegia (HSP) or spastic paraplegia related to the Kennedy pathway. This condition affects the nervous system, particularly the long nerve fibers that run down the spinal cord and control movement in the legs. When these nerve fibers are damaged or do not work properly, the muscles in the legs become stiff and weak over time. The Kennedy pathway is a set of chemical reactions inside cells that helps build import

How is Autosomal recessive complex spastic paraplegia due to Kennedy pathway dysfunction inherited?

Autosomal recessive complex spastic paraplegia due to Kennedy pathway dysfunction follows a autosomal recessive inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.