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Advances of pharmacological therapies in lipodystrophy syndromes: current evidence and future directions

Ozge Besci1, Merve Celik Guler1, Maria Cristina Foss-Freitas1

  • 1Division of Metabolism, Endocrinology and Diabetes, Department of Internal Medicine, University of Michigan Medical School, Ann Arbor, MI, USA.

Expert Review of Endocrinology & Metabolism
|November 5, 2025
PubMed
Abstract

Insights

Lipodystrophy syndromes involve adipose tissue loss and metabolic complications. Current management is symptomatic, but emerging therapies offer hope for improved long-term outcomes in this rare disorder.

Area of Science:

  • Rare genetic and acquired disorders
  • Adipose tissue biology and metabolism
  • Endocrinology and metabolic diseases

Background:

  • Lipodystrophy syndromes are rare, heterogeneous disorders.
  • Characterized by partial or generalized loss of adipose tissue.
  • Associated with ectopic fat deposition and severe metabolic complications.

Purpose of the Study:

  • To review current and emerging therapies for lipodystrophy syndromes.
  • To discuss the management strategies for this complex group of disorders.
  • To highlight unmet clinical needs and future therapeutic directions.

Main Methods:

  • Comprehensive literature search of available databases.
  • Identification and analysis of studies on lipodystrophy syndrome therapies.
  • Synthesis of information on current and novel treatment approaches.

Main Results:

  • Current management relies on dietary modification, exercise, lifestyle changes, and metreleptin therapy.
  • Conventional therapies are employed to manage specific metabolic complications.
  • Novel interventions are under investigation to address the limitations of current treatments.

Conclusions:

  • No definitive cure currently exists for lipodystrophy syndromes.
  • Symptomatic management is the current standard of care.
  • Emerging therapies hold promise for expanding treatment options and improving patient outcomes.
Keywords:
Adeno-associated viral vectorsCRISPR-Cas9DNA TransferFGF-21Hutchinson-Gilford progeria syndromegene editinglipodystrophymetreleptinmibavademabtirzepatide

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