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Case Report: Twenty years of metreleptin therapy in congenital generalized lipodystrophy type 1: the longest reported follow-up to date.

Frontiers in endocrinology·2026
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Clinical Guidelines for the Diagnosis and Management of Generalized and Partial Lipodystrophies: A Position Statement Endorsed by the Hellenic Endocrine Society.

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Metabolic effects of pioglitazone and sodium-glucose cotransporter 2 inhibitors in familial partial lipodystrophy.

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Update on Therapeutic Options in Lipodystrophy.

Baris Akinci1,2, Rasimcan Meral1, Elif Arioglu Oral3

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Metreleptin therapy offers significant benefits for metabolic abnormalities in lipodystrophy syndromes. This review details current treatments and investigational therapies for generalized and partial lipodystrophy.

Keywords:
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Area of Science:

  • Endocrinology
  • Metabolic Disorders
  • Pharmacology

Background:

  • Lipodystrophy syndromes are characterized by leptin deficiency and associated metabolic abnormalities.
  • Leptin replacement therapy has shown promise in animal models and human studies.
  • Recent approvals of metreleptin mark a significant advancement in treatment.

Purpose of the Study:

  • To review conventional and metreleptin therapy for lipodystrophy syndromes.
  • To highlight current investigational treatments for lipodystrophy.
  • To provide a comprehensive overview of therapeutic approaches.

Main Methods:

  • Literature review of studies on lipodystrophy treatment.
  • Analysis of clinical trial data for metreleptin.
  • Summary of ongoing research for novel therapies.

Main Results:

  • Metreleptin demonstrates significant benefits in treating metabolic abnormalities in generalized lipodystrophy.
  • Metreleptin is approved for generalized lipodystrophy (FDA) and both generalized and partial lipodystrophy (EMA).
  • Current treatment for generalized lipodystrophy involves metreleptin as an adjunct to diet and standard care.

Conclusions:

  • Metreleptin is a key therapeutic option for generalized lipodystrophy.
  • Research is ongoing to develop novel treatments, particularly for partial lipodystrophy.
  • A multi-faceted approach combining established and investigational therapies is crucial for managing lipodystrophy syndromes.