Safety and efficacy of N-acetylmannosamine (ManNAc) in patients with GNE myopathy: an open-label phase 2 study
N-乙酰甘露糖胺 (ManNAc) 治疗 GNE 肌病患者的安全性和有效性:一项开放标签的 II 期研究
期刊:Genetics in Medicine
影响因子:6.2
doi:10.1038/s41436-021-01259-x
Carrillo, Nuria; Malicdan, May C; Leoyklang, Petcharat; Shrader, Joseph A; Joe, Galen; Slota, Christina; Perreault, John; Heiss, John D; Class, Bradley; Liu, Chia-Ying; Bradley, Kennan; Jodarski, Colleen; Ciccone, Carla; Driscoll, Claire; Parks, Rebecca; Van Wart, Scott; Bayman, Levent; Coffey, Christopher S; Quintana, Melanie; Berry, Scott M; Huizing, Marjan; Gahl, William A