title: Metreleptin for injection to treat the complications of leptin deficiency in patients with congenital or acquired generalized lipodystrophy.
abstract: The lipodystrophies represent a class of diseases characterized by leptin deficiency. Leptin deficiency is associated with a severe form of the metabolic syndrome characterized by dyslipidemia, insulin resistance, diabetes, and ovarian dysfunction. Metreleptin is the pharmaceutical derived product that has been approved by the Food and Drug Administration (FDA) to treat the severe metabolic abnormalities of the generalized forms of lipodystrophy. Herein we describe the properties of metreleptin, its use in patients, which includes the administration of the drug and how it may be acquired by medical professionals as well as its safety, tolerability, and properties. Finally, we speculate on future uses and development of metreleptin.
authors.affiliation: a Diabetes, Endocrinology, and Obesity Branch , National Institute of Diabetes and Digestive Kidney Diseases, National Institutes of Health , Bethesda , MD , USA.
authors.forename: Cristina Adelia
authors.initials: CA
authors.lastname: Meehan
authors.affiliation: a Diabetes, Endocrinology, and Obesity Branch , National Institute of Diabetes and Digestive Kidney Diseases, National Institutes of Health , Bethesda , MD , USA.
authors.forename: Elaine
authors.initials: E
authors.lastname: Cochran
authors.affiliation: a Diabetes, Endocrinology, and Obesity Branch , National Institute of Diabetes and Digestive Kidney Diseases, National Institutes of Health , Bethesda , MD , USA.
authors.forename: Andrea
authors.initials: A
authors.lastname: Kassai
authors.affiliation: a Diabetes, Endocrinology, and Obesity Branch , National Institute of Diabetes and Digestive Kidney Diseases, National Institutes of Health , Bethesda , MD , USA.
authors.forename: Rebecca J
authors.initials: RJ
authors.lastname: Brown
authors.affiliation: a Diabetes, Endocrinology, and Obesity Branch , National Institute of Diabetes and Digestive Kidney Diseases, National Institutes of Health , Bethesda , MD , USA.
authors.forename: Phillip
authors.initials: P
authors.lastname: Gorden
mesh_terms: D006801:Humans; D007267:Injections; D020738:Leptin / Q000008:administration & dosage / Q000009:adverse effects / Q000031:analogs & derivatives* / Q000172:deficiency / Q000627:therapeutic use; D008060:Lipodystrophy / Q000188:drug therapy* / Q000503:physiopathology; D052497:Lipodystrophy, Congenital Generalized / Q000188:drug therapy* / Q000503:physiopathology; D012720:Severity of Illness Index
chemical_list: D020738:Leptin; C415771:metreleptin
keywords: insulin resistance; leptin; lipodystrophy; metabolic syndrome; metreleptin