Laminopathy with lipodystrophy

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ORPHA:300763
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9Specialists8Treatment centers

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Overview

Laminopathy with lipodystrophy is a rare genetic condition that affects the body's fat tissue distribution and can also impact muscles, the heart, and metabolism. It belongs to a group of diseases called laminopathies, which are caused by problems in genes that produce proteins forming the structural framework of cell nuclei — the control centers inside every cell. In this condition, the body loses fat from certain areas (such as the arms, legs, and face) while sometimes gaining excess fat in other areas (like the neck or trunk). This abnormal fat distribution is called lipodystrophy. Because fat tissue plays an important role in metabolism, people with this condition often develop insulin resistance, diabetes, high triglyceride levels, and fatty liver disease. Some patients may also experience muscle weakness, heart problems (such as cardiomyopathy or abnormal heart rhythms), and skin changes. The severity can vary widely from person to person. Treatment focuses on managing metabolic complications like diabetes and high cholesterol, protecting the heart, and addressing other symptoms as they arise. There is currently no cure, but careful medical management can help improve quality of life and reduce the risk of serious complications. The condition may also be referred to as LMNA-related lipodystrophy or familial partial lipodystrophy type 2 (FPLD2), depending on the specific genetic cause and clinical presentation.

Key symptoms:

Loss of fat under the skin in the arms, legs, and buttocksExcess fat around the neck, face, or trunkInsulin resistance or type 2 diabetesHigh triglyceride levels in the bloodFatty liver diseaseMuscle weaknessAbnormal heart rhythmsEnlarged or weakened heart (cardiomyopathy)Darkened skin patches (acanthosis nigricans)Irregular menstrual periods in womenPolycystic ovary syndrome (PCOS) in womenEarly heart diseaseJoint stiffness or contracturesProminent veins due to lack of fat under the skin

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 lipodystrophy.

View clinical trials →

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

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Search ClinicalTrials.gov ↗Join the Laminopathy with lipodystrophy community →

Specialists

9 foundView all specialists →
DS
David Savage
Specialist
PI on 1 active trial
EM
Elif A Oral, M.D.
ANN ARBOR, MI
Specialist
PI on 1 active trial

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 lipodystrophy.

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Community

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Latest news about Laminopathy with lipodystrophy

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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 gene mutation do I have, and how does it affect my prognosis?,How often should I have my heart checked with echocardiograms and ECGs?,Am I a candidate for metreleptin (Myalept) therapy?,What dietary changes are most important for managing my triglycerides and blood sugar?,Should my family members be tested for this genetic condition?,What are the warning signs of a cardiac emergency I should watch for?,Are there any clinical trials or new treatments I should know about?

Common questions about Laminopathy with lipodystrophy

What is Laminopathy with lipodystrophy?

Laminopathy with lipodystrophy is a rare genetic condition that affects the body's fat tissue distribution and can also impact muscles, the heart, and metabolism. It belongs to a group of diseases called laminopathies, which are caused by problems in genes that produce proteins forming the structural framework of cell nuclei — the control centers inside every cell. In this condition, the body loses fat from certain areas (such as the arms, legs, and face) while sometimes gaining excess fat in other areas (like the neck or trunk). This abnormal fat distribution is called lipodystrophy. Because

How is Laminopathy with lipodystrophy inherited?

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

Which specialists treat Laminopathy with lipodystrophy?

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