转甲状腺素
医学
安慰剂
内科学
心肌病
心脏病学
淀粉样变性
置信区间
胃肠病学
泌尿科
内分泌学
心力衰竭
病理
替代医学
作者
Julian D. Gillmore,Daniel P. Judge,Francesco Cappelli,Marianna Fontana,Pablo García‐Pavía,Simon Gibbs,Martha Grogan,Mazen Hanna,James E. Hoffman,Ahmad Masri,Mathew S. Maurer,José Nativi-Nicolau,Laura Obici,Steen Hvitfeldt Poulsen,Frank W. Rockhold,Keyur B. Shah,Prem Soman,Jyotsna Garg,Karen Chiswell,Haolin Xu
标识
DOI:10.1056/nejmoa2305434
摘要
Transthyretin amyloid cardiomyopathy is characterized by the deposition of misfolded monomeric transthyretin (TTR) in the heart. Acoramidis is a high-affinity TTR stabilizer that acts to inhibit dissociation of tetrameric TTR and leads to more than 90% stabilization across the dosing interval as measured ex vivo.
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