Once-weekly TransCon CNP (navepegritide) in children with achondroplasia (ACcomplisH): a phase 2, multicentre, randomised, double-blind, placebo-controlled, dose-escalation trial
每周一次TransCon CNP(navepegritide)治疗软骨发育不全患儿(ACcomplisH):一项II期、多中心、随机、双盲、安慰剂对照、剂量递增试验
期刊:EClinicalMedicine
影响因子:10
doi:10.1016/j.eclinm.2023.102258
Savarirayan, Ravi; Hoernschemeyer, Daniel G; Ljungberg, Merete; Zarate, Yuri A; Bacino, Carlos A; Bober, Michael B; Legare, Janet M; Högler, Wolfgang; Quattrin, Teresa; Abuzzahab, M Jennifer; Hofman, Paul L; White, Klane K; Ma, Nina S; Schnabel, Dirk; Sousa, Sérgio B; Mao, Meng; Smith, Alden; Chakraborty, Mukta; Giwa, Adebola; Winding, Bent; Volck, Birgitte; Shu, Aimee D; McDonnell, Ciara