Two decades of experience of the Fabry Outcome Survey provides further confirmation of the long-term effectiveness of agalsidase alfa enzyme replacement therapy
二十年来，Fabry 结局调查的经验进一步证实了阿加糖酶α酶替代疗法的长期疗效。
期刊:Molecular Genetics and Metabolism Reports
影响因子:1.9
doi:10.1016/j.ymgmr.2025.101215
Ramaswami, Uma; Pintos-Morell, Guillem; Kampmann, Christoph; Nicholls, Kathleen; Niu, Dau-Ming; Reisin, Ricardo; West, Michael L; Anagnostopoulou, Christina; Botha, Jaco; Jazukeviciene, Dalia; Schenk, Jörn; Hughes, Derralynn A; Giugliani, Roberto
