Venglustat, an orally administered glucosylceramide synthase inhibitor: Assessment over 3 years in adult males with classic Fabry disease in an open-label phase 2 study and its extension study
Venglustat,一种口服葡糖基神经酰胺合成酶抑制剂:一项开放标签的 II 期研究及其扩展研究,对患有经典法布里病的成年男性进行了为期 3 年的评估。
期刊:Molecular Genetics and Metabolism
影响因子:3.5
doi:10.1016/j.ymgme.2022.11.002
Deegan, Patrick B; Goker-Alpan, Ozlem; Geberhiwot, Tarekegn; Hopkin, Robert J; Lukina, Elena; Tylki-Szymanska, Anna; Zaher, Atef; Sensinger, Charlotte; Gaemers, Sebastiaan J M; Modur, Vijay; Thurberg, Beth L; Sharma, Jyoti; Najafian, Behzad; Mauer, Michael; DasMahapatra, Pronabesh; Wilcox, William R; Germain, Dominique P