【摘要】 patisiran是一种包裹在脂质纳米粒子中的双链小干扰RNA(siRNA),通过降低血清遗传性转甲状腺素(TTR)蛋白水平,从而稳定或改善神经病变。已于2018年8月相继被FDA和欧洲药品管理局(EMA)批准,用于TTR介导的淀粉样变性病(hATTR)Ⅰ期或Ⅱ期多发性神经病变成人患者的治疗。本文主要从该药品的作用机制、药效学、药动学、临床试验、用法用量及相关的不良反应等几个方面进行阐述。更多还原

【Abstract】 Patisiran is a double-stranded siRNA encapsulated in lipid nanoparticles that stabilizes or improves neuropathy by lowering serum hereditary transthyretin(TTR) protein levels. Patisiran was approved by FDA and the European Medicines Agency(EMA) in August 2018 for treatment of TTR-mediated amyloidosis(hATTR) stage Ⅰ or adult patients with stage Ⅱ polyneuropathy. This paper mainly describes the mechanism of action,pharmacodynamics,pharmacokinetics,clinical trials,usage and dosage,and related adverse reactions of patisiran.更多还原