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Lipodystrophy United
Lipodystrophy United
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  • More
    • Home
    • About LU
      • About Us
      • Schedule of Events
      • Our History
      • Our Team
      • LU Symposia
      • World Lipodystrophy Day
    • What is Lipodystrophy
      • The Basics
      • Types
      • Research and News
    • Patient Corner
      • Guides to Lipodystrophy
      • Stories & Community
      • Expert Centers
      • Lectures
      • Clinical Trials
      • Rare Disease Resources
    • Get Involved
      • Donate
      • Volunteer
      • Engagement Opportunities
  • Home
  • About LU
    • About Us
    • Schedule of Events
    • Our History
    • Our Team
    • LU Symposia
    • World Lipodystrophy Day
  • What is Lipodystrophy
    • The Basics
    • Types
    • Research and News
  • Patient Corner
    • Guides to Lipodystrophy
    • Stories & Community
    • Expert Centers
    • Lectures
    • Clinical Trials
    • Rare Disease Resources
  • Get Involved
    • Donate
    • Volunteer
    • Engagement Opportunities

Lipodystrophy Studies

Oral Glucagon-Like Peptide 1 Analog as an Adjuvant Therapy in Generalized Lipodystrophy

*The Diagnosis and Management of Lipodystrophy Syndromes: A Multi-Society Practice Guideline*

Oral Glucagon-Like Peptide 1 Analog as an Adjuvant Therapy in Generalized Lipodystrophy

 A new case study co-authored by Lipodystrophy United’s Scientific Director, Sharon Halperin, highlights the first successful use of an oral GLP-1 medication (semaglutide) in a patient with generalized lipodystrophy (GL). After starting the medication, the patient was able to stop insulin completely while maintaining excellent blood sugar control. This research shows promising potential for oral GLP-1 therapies to improve treatment options and quality of life for people living with GL. Lipodystrophy United is proud to help lead research driving innovation in the field. 

Full Paper Here

Assessing the Burden of Lipodystrophy: Insights from a Patient-Driven Survey

*The Diagnosis and Management of Lipodystrophy Syndromes: A Multi-Society Practice Guideline*

Oral Glucagon-Like Peptide 1 Analog as an Adjuvant Therapy in Generalized Lipodystrophy

 Lipodystrophy United remains committed to elevating the voices of our community. In our most recent abstract, we share key findings from a patient survey exploring the burden of living with lipodystrophy. Responses from 48 individuals revealed widespread challenges, including pain, fatigue, hunger, body image issues, mental health concerns, and less commonly recognized symptoms such as muscle weakness and brain fog. This work has been accepted for presentation at several major conferences in 2025, including ENDO 2025—the world’s premier endocrinology meeting.

Full Abstract Here

*The Diagnosis and Management of Lipodystrophy Syndromes: A Multi-Society Practice Guideline*

*The Diagnosis and Management of Lipodystrophy Syndromes: A Multi-Society Practice Guideline*

*The Diagnosis and Management of Lipodystrophy Syndromes: A Multi-Society Practice Guideline*

Send this paper to your doctor and review it thoroughly!

Patients should get regular checkups for diabetes, high cholesterol, and other health issues. A proper diet is crucial for managing these problems. Metreleptin therapy works well for some patients with low leptin levels and certain types of lipodystrophy. Other general treatments like metformin for diabetes and statins or fibrates for high cholesterol may also help. 

Full Paper Here

Impact of lipodystrophy on health-related quality of life

Lipodystrophy: a paradigm for understanding the consequences of “overloading” adipose tissue

*The Diagnosis and Management of Lipodystrophy Syndromes: A Multi-Society Practice Guideline*

Over a 24-month period, 27.69% of adult patients were diagnosed with various psychiatric disorders, such as depressive episodes, anxiety disorders, and adjustment disorders, highlighting underdiagnosis of psychiatric disorders among lipodystrophy patients.

Full Paper Here

Lipodystrophy: a paradigm for understanding the consequences of “overloading” adipose tissue

Lipodystrophy: a paradigm for understanding the consequences of “overloading” adipose tissue

Lipodystrophy: a paradigm for understanding the consequences of “overloading” adipose tissue

The paper discusses the various roles of adipose tissue. Leptin helps regulate our eating habits and energy usage, and a lack of it can lead to overeating and obesity. Ectopic fat accumulation (fat in the wrong places) is a common feature of lipodystrophies, and theories have been proposed to understand why it is linked to insulin resistance. 

Full Paper here

Diagnosis and treatment of lipodystrophy: a step-by-step approach

Lipodystrophy: a paradigm for understanding the consequences of “overloading” adipose tissue

Lipodystrophy: a paradigm for understanding the consequences of “overloading” adipose tissue

Lipodystrophy leads to metabolic issues like diabetes and high cholesterol. This article aims to help doctors identify and treat lipodystrophy by providing steps for diagnosis and management. Lipodystrophy can be genetic or acquired, and it can affect the entire body or just parts of it. Recognizing lipodystrophy is crucial as it can be misdiagnosed or overlooked.

Full Paper Here

"Fat Shadows" From DXA for the Qualitative Assessment of Lipodystrophy

"Fat Shadows" From DXA for the Qualitative Assessment of Lipodystrophy

"Fat Shadows" From DXA for the Qualitative Assessment of Lipodystrophy

The study introduces a diagnostic method called "fat shadows" derived from DXA scans to identify lipodystrophy syndromes

Full paper here

Patients as Partners in Rare Disease Diagnosis and Research

"Fat Shadows" From DXA for the Qualitative Assessment of Lipodystrophy

"Fat Shadows" From DXA for the Qualitative Assessment of Lipodystrophy

Patients' direct experiences have been crucial in shaping the work of clinicians and researchers, bringing valuable perspectives to the forefront. 

Full Paper Here

Effects of Metreleptin on Patient Outcomes and Quality of Life

"Fat Shadows" From DXA for the Qualitative Assessment of Lipodystrophy

Effects of Metreleptin on Patient Outcomes and Quality of Life

Metreleptin is associated with meaningful clinical and quality-of-life enhancements for individuals with both lipodystrophy.

Full Paper Here

Lipodystrophy in the news

Delayed Diagnoses and Misunderstood Symptoms: The challenge of rare disease Lipodystrophy March 26,

Advocates Andra Stratton and Sharon Halperin are raising awareness of lipodystrophy, a rare fat disorder often misdiagnosed; through global networks, they aim to improve diagnosis, access to care, and support for patients in the US and Israel.

Read Here

Pioneering Gene Therapy Research Offers New Hope for Lipodystrophy

A groundbreaking gene therapy project led by Dr. George McIlroy and Prof. Justin Rochford is gaining momentum in the fight against lipodystrophy. Selected for the prestigious Scottish Enterprise and Cell and Gene Therapy Catapult workshop, the team is working to develop a Target Product Profile (TPP) for a potential treatment that targets the root cause of this rare and debilitating condition. Their innovative approach could transform the lives of patients by restoring healthy fat tissue function and dramatically improving long-term outcomes.

Read Here

Entos Pharmaceuticals Awarded $4 Million USD in Funding

Entos Pharmaceuticals has received a $4 million grant from the California Institute for Regenerative Medicine (CIRM) to advance ENTLEP001, a promising investigational gene therapy for congenital generalized lipodystrophy (CGL). ENTLEP001 aims to provide durable, redosable leptin expression using Entos’ proprietary Fusogenix™ PLV™ delivery platform. This new approach could significantly improve outcomes and reduce treatment burden compared to current therapies like metreleptin. The funding will support key preclinical activities needed to move the therapy closer to clinical trials.

Read Here

Global Genes: Health Canada Approves Chiesi’s Myalept for Lipodystrophy Feb. 5, 2024

Health Canada has approved Chiesi Global Rare Diseases' Myalept as an adjunct to diet for treating complications of leptin deficiency in lipodystrophy. The approval covers patients with congenital or acquired generalized lipodystrophy and familial partial lipodystrophy who haven't responded to standard treatments. Myalept, developed by Amryt Pharma and acquired by Chiesi Group, showed significant efficacy in reducing metabolic abnormalities in both generalized and partial lipodystrophy patients, with common adverse reactions including weight loss, hypoglycemia, and fatigue.

Read Here

Fred Hutch Cancer Center, Using a mouse model to combat the effects of lipodystrophy Oct. 16, 2023

Through mouse models, researchers discovered that enhancing NRF2 expression, a transcription factor regulating various cellular pathways including oxidative stress response, in the liver can protect against lipodystrophy-induced hepatic damage. Their research, recently published in the International Journal of Molecular Sciences, demonstrates that liver-directed NRF2 targeting could offer therapeutic opportunities to mitigate the effects of lipodystrophy, potentially preventing fatty liver disease and other metabolic complications associated with the condition.

Read Here

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EIN: 46-1165699


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