The Lancet· Riccardo Papa, Susan Rhee, Patricia Delai, Keqin Zhang, Ignacio Briceño Balcázar, Kavitha Rethanavelu, Toshifumi Fujiwara, Kenichi Mishima, Jacek Tabarkiewicz, Thomas Funck-Brentano, Raphaella Stander, Javier Bachiller-Corral, Kathryn M Dahir, E Marelise W Eekhoff, Matti Hero, Evelyn Kuong, Naoto Uemura, Meijian Zhou, Dinko Gonzalez Trotter, Jing Gu, Robert J Sanchez, Donal Óg O’Donovan, Aris N Economides, Robert Pordy, Boaz Hirshberg, George D Yancopoulos, Richard Keen, OPTIMA Study Group·· 3 天前精选评分64
garetosmab 在 FOP 三期试验 OPTIMA 中显著减少新异位骨病变
[Articles] Efficacy and safety of garetosmab, an activin A-blocking antibody, in fibrodysplasia ossificans progressiva (OPTIMA): a randomised, double-blind, placebo-controlled, phase 3 trial
导读
garetosmab（激活素 A 阻断抗体）在 OPTIMA 三期随机双盲安慰剂对照试验中显著减少进行性骨化性纤维发育不良（FOP）患者的新异位骨病变数量，提供了其作为有效且耐受良好的疾病修饰疗法的证据。
推荐理由
原文提供了三期试验中异位骨病变减少的关键数据，读者可据此评估该抗体对进行性骨化性纤维发育不良的疾病修饰潜力。
来源：The Lancet · thelancet.com