Clinical outcomes after 4.5 years of eliglustat therapy for Gaucher disease type 1: Phase 3 ENGAGE trial final results
接受eliglustat治疗4.5年后戈谢病1型患者的临床结果:3期ENGAGE试验的最终结果
期刊:American Journal of Hematology
影响因子:9.9
doi:10.1002/ajh.26276
Mistry, Pramod K; Lukina, Elena; Ben Turkia, Hadhami; Shankar, Suma P; Baris Feldman, Hagit; Ghosn, Marwan; Mehta, Atul; Packman, Seymour; Lau, Heather; Petakov, Milan; Assouline, Sarit; Balwani, Manisha; Danda, Sumita; Hadjiev, Evgueniy; Ortega, Andres; Foster, Meredith C; Gaemers, Sebastiaan J M; Peterschmitt, M Judith