首页 正文

Patisiran for the Treatment of Transthyretin-mediated Amyloidosis with Cardiomyopathy

{{output}}
Transthyretin (TTR) is a tetrameric protein, synthesized primarily by the liver, that acts as a physiological transport protein for retinol and thyroxine. TTR can misfold into pathogenic amyloid fibrils that deposit in the heart and nerves, causing a life-thre... ...