Laminopathy with peripheral neuropathy

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ORPHA:300758
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1Specialists8Treatment centers

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UniteRare data is sourced from FDA.gov, ClinicalTrials.gov, Orphanet, OMIM, and NORD.
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Overview

Laminopathy with peripheral neuropathy is an extremely rare genetic disorder caused by mutations in genes that produce lamin proteins, which are essential building blocks of the structure surrounding the cell nucleus (called the nuclear envelope). Lamins help maintain the shape and function of cells throughout the body. When these proteins do not work properly, multiple tissues can be affected. In this particular form of laminopathy, the hallmark feature is peripheral neuropathy — damage to the nerves that carry signals between the brain, spinal cord, and the rest of the body. This can lead to numbness, tingling, pain, and weakness, especially in the hands and feet. Patients may also experience muscle wasting and difficulty with movement over time. Some individuals may have additional features seen in other laminopathies, such as mild muscle weakness in the limbs or subtle changes in fat distribution. Because this condition is so rare, treatment is mainly supportive and focused on managing symptoms. There is no cure at this time. Physical therapy, pain management, and assistive devices may help maintain function and quality of life. Ongoing research into laminopathies may eventually lead to more targeted treatments.

Key symptoms:

Numbness or tingling in hands and feetMuscle weakness, especially in the arms and legsMuscle wasting over timePain in the extremitiesDifficulty walking or balance problemsReduced reflexesLoss of sensation in the feet or handsFatigueDifficulty with fine motor tasks like buttoning clothesFoot drop or abnormal gait

Inheritance

Autosomal dominant

Passed on from just one parent; each child has about a 50% chance of inheriting it

Age of Onset

Variable

Can begin at different ages, from infancy through adulthood

Orphanet ↗NORD ↗

Treatments

No FDA-approved treatments are currently listed for Laminopathy with peripheral neuropathy.

View clinical trials →

No actively recruiting trials found for Laminopathy with peripheral neuropathy at this time.

New trials open frequently. Follow this disease to get notified.

Search ClinicalTrials.gov ↗Join the Laminopathy with peripheral neuropathy community →

Specialists

1 foundView all specialists →
EP
Elena Biagini, MD, PhD
Chieti, Abruzzo/Chieti
Specialist

Rare Disease Specialist

PI on 2 active trials

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 Laminopathy with peripheral neuropathy.

Search all travel grants →NORD Financial Assistance ↗

Community

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Latest news about Laminopathy with peripheral neuropathy

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Caregiver Resources

NORD Caregiver Resources

Support, advocacy, and financial assistance for caregivers of rare disease patients.

Mental Health Support

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Family & Caregiver Grants

Financial assistance programs specifically for caregivers of rare disease patients.

Social Security Disability

Learn how rare disease patients may qualify for SSDI/SSI benefits.

Questions for your doctor

Bring these to your next appointment

  • Q1.What specific LMNA mutation do I have, and what does it mean for my health?,How quickly might my symptoms progress over time?,Do I need regular heart monitoring, and how often?,What therapies or medications can help manage my nerve pain and weakness?,Are there any clinical trials or research studies I could participate in?,Should my family members be tested for this genetic change?,What specialists should I see regularly, and how often?

Common questions about Laminopathy with peripheral neuropathy

What is Laminopathy with peripheral neuropathy?

Laminopathy with peripheral neuropathy is an extremely rare genetic disorder caused by mutations in genes that produce lamin proteins, which are essential building blocks of the structure surrounding the cell nucleus (called the nuclear envelope). Lamins help maintain the shape and function of cells throughout the body. When these proteins do not work properly, multiple tissues can be affected. In this particular form of laminopathy, the hallmark feature is peripheral neuropathy — damage to the nerves that carry signals between the brain, spinal cord, and the rest of the body. This can lead t

How is Laminopathy with peripheral neuropathy inherited?

Laminopathy with peripheral neuropathy follows a autosomal dominant inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.

Which specialists treat Laminopathy with peripheral neuropathy?

1 specialists and care centers treating Laminopathy with peripheral neuropathy are listed on UniteRare, sourced from ClinicalTrials.gov principal investigators, published research, and the NPPES NPI registry.