Lucerastat, an oral therapy for Fabry disease: results from a pivotal randomized phase 3 study and its open-label extension
Lucerastat,一种用于治疗法布里病的口服药物:一项关键性随机3期研究及其开放标签扩展研究的结果
期刊:Nature Communications
影响因子:15.7
doi:10.1038/s41467-025-68256-5
Nordbeck, Peter; Goker-Alpan, Ozlem; Bernat, John A; Germain, Dominique P; Giraldo, Pilar; Jovanovic, Ana; Kimonis, Virginia; Nicholls, Kathleen; Rockman-Greenberg, Cheryl; Schiffmann, Raphael; Thomas, Mark; Tylki-Szymanska, Anna; Wallace, Eric; Welford, Richard W D; West, Michael L; Clozel, Martine; Frey, Aline; Trokan, Luba; Mueller, Markus S; Vogler, Markus; Wanner, Christoph; Hughes, Derralynn