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注射用美曲普汀用于治疗先天性或获得性全身性脂肪营养不良患者的瘦素缺乏并发症。

Metreleptin for injection to treat the complications of leptin deficiency in patients with congenital or acquired generalized lipodystrophy.

作者信息

Meehan Cristina Adelia, Cochran Elaine, Kassai Andrea, Brown Rebecca J, Gorden Phillip

机构信息

a Diabetes, Endocrinology, and Obesity Branch , National Institute of Diabetes and Digestive Kidney Diseases, National Institutes of Health , Bethesda , MD , USA.

出版信息

Expert Rev Clin Pharmacol. 2016;9(1):59-68. doi: 10.1586/17512433.2016.1096772. Epub 2015 Oct 14.

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.

摘要

脂肪营养不良是一类以瘦素缺乏为特征的疾病。瘦素缺乏与一种严重形式的代谢综合征相关,该综合征的特征为血脂异常、胰岛素抵抗、糖尿病和卵巢功能障碍。美曲普明是一种已获美国食品药品监督管理局(FDA)批准用于治疗全身性脂肪营养不良严重代谢异常的药物衍生产品。在此我们描述美曲普明的特性、其在患者中的应用,包括药物的给药方式以及医学专业人员获取该药物的途径,还有其安全性、耐受性和特性。最后,我们推测美曲普明的未来用途和发展。

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