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

INTRODUCTION: Lipodystrophy syndromes are a heterogeneous group of rare disorders characterized by partial or generalized loss of adipose tissue, which may be either inherited or acquired. Loss of adipose tissue in typical storage sites starting from birth or later in life, combined with abnormal fat accumulation in other organs, contributes to multiple metabolic complications. There is currently no definitive cure available for lipodystrophy syndromes, and clinical management remains symptomatic.

AREAS COVERED: For this review, available databases were searched to identify publications and studies on current and emerging therapies to discuss the management of lipodystrophy syndromes. Dietary modification, exercise, lifestyle management, and metreleptin therapy are the mainstay of treatment, while conventional therapies are used to target specific complications. Novel interventions are under investigation to address unmet clinical needs.

EXPERT OPINION: There is currently no cure for lipodystrophy syndromes. Emerging therapies are being investigated to expand therapeutic options and improve long-term outcomes of this complex disorder.