Survival in infants treated with sebelipase Alfa for lysosomal acid lipase deficiency: an open-label, multicenter, dose-escalation study
使用塞贝利酶α治疗溶酶体酸性脂肪酶缺乏症婴儿的生存率：一项开放标签、多中心、剂量递增研究
期刊:Orphanet Journal of Rare Diseases
影响因子:3.5
doi:10.1186/s13023-017-0587-3
Jones, Simon A; Rojas-Caro, Sandra; Quinn, Anthony G; Friedman, Mark; Marulkar, Sachin; Ezgu, Fatih; Zaki, Osama; Gargus, J Jay; Hughes, Joanne; Plantaz, Dominique; Vara, Roshni; Eckert, Stephen; Arnoux, Jean-Baptiste; Brassier, Anais; Le Quan Sang, Kim-Hanh; Valayannopoulos, Vassili
