A multi-country time and motion study to describe the experience and burden associated with the treatment of Fabry disease with enzyme replacement therapy with agalsidase alfa and agalsidase beta
一项多国时间动作研究,旨在描述使用阿加糖酶α和阿加糖酶β进行酶替代疗法治疗法布里病患者的经验和相关负担。
期刊:Orphanet Journal of Rare Diseases
影响因子:3.5
doi:10.1186/s13023-025-03707-2
Keyzor, Ian; Martins, Ana Maria; Uçar, Sema Kalkan; Yamakawa, Hiroyuki; Chien, Yin-Hsiu; Arslan, Nur; Niu, Dau-Ming; Tümer, Leyla; Baldock, Laura; Shohet, Simon; Giuliano, Joseph D