Multi-Year Registry Study of Elapegademase Treatment in Patients With Adenosine Deaminase Severe Combined Immunodeficiency (ADA-SCID) Requiring Enzyme Replacement Therapy
一项针对需要酶替代疗法的腺苷脱氨酶重症联合免疫缺陷病（ADA-SCID）患者的Elapegademase治疗多年注册研究
期刊:Journal of Clinical Immunology
影响因子:5.7
doi:10.1007/s10875-025-01873-3
Dorsey, Morna J; Butte, Manish J; Lieberman, Jay A; Lehman, Heather; Fausnight, Tracy; Keller, Michael D; Fradette, Caroline; Hershfield, Michael S; Pozos, Tamara C; Rozova, Anna; Wall, Luke A; Bednarski, Jeffrey J; Tarrant, Teresa K; Chong, Hey J; Geng, Bob; Temin, Noemi Toiber; Laubach, Susan S; Lin, Leo; Mousallem, Talal; Walter, Jolan E
