日期:
2020 年 — 2026 年
2020
2021
2022
2023
2024
2025
2026
影响因子:

Neuroimaging findings in infantile Pompe patients treated with enzyme replacement therapy

接受酶替代疗法治疗的婴儿型庞贝病患者的神经影像学发现

McIntosh, Paul T; Hobson-Webb, Lisa D; Kazi, Zoheb B; Prater, Sean N; Banugaria, Suhrad G; Austin, Stephanie; Wang, Raymond; Enterline, David S; Frush, Donald P; Kishnani, Priya S

Enhanced efficacy from gene therapy in Pompe disease using coreceptor blockade

利用辅助受体阻断增强基因疗法在庞贝病中的疗效

Han, Sang-oh; Li, Songtao; Brooks, Elizabeth D; Masat, Elisa; Leborgne, Christian; Banugaria, Suhrad; Bird, Andrew; Mingozzi, Federico; Waldmann, Herman; Koeberl, Dwight

Letter to the Editors: Concerning "CRIM-negative Pompe disease patients with satisfactory clinical outcomes on enzyme replacement therapy" by Al Khallaf et al

致编辑的信:关于 Al Khallaf 等人发表的题为“CRIM 阴性庞贝病患者接受酶替代疗法后获得满意的临床结果”的文章

Prater, Sean N; Banugaria, Suhrad G; Morgan, Claire; Sung, Crystal C; Rosenberg, Amy S; Kishnani, Priya S

Non-depleting anti-CD4 monoclonal antibody induces immune tolerance to ERT in a murine model of Pompe disease

非耗竭性抗CD4单克隆抗体在庞贝病小鼠模型中诱导对酶替代疗法的免疫耐受

Sun, Baodong; Banugaria, Suhrad G; Prater, Sean N; Patel, Trusha T; Fredrickson, Keri; Ringler, Douglas J; de Fougerolles, Antonin; Rosenberg, Amy S; Waldmann, Herman; Kishnani, Priya S

Bortezomib in the rapid reduction of high sustained antibody titers in disorders treated with therapeutic protein: lessons learned from Pompe disease

硼替佐米在快速降低接受治疗性蛋白治疗的疾病中持续高抗体滴度方面的作用:从庞贝病中汲取的经验教训

Banugaria, Suhrad G; Prater, Sean N; McGann, Judeth K; Feldman, Jonathan D; Tannenbaum, Jesse A; Bailey, Carrie; Gera, Renuka; Conway, Robert L; Viskochil, David; Kobori, Joyce A; Rosenberg, Amy S; Kishnani, Priya S

Algorithm for the early diagnosis and treatment of patients with cross reactive immunologic material-negative classic infantile pompe disease: a step towards improving the efficacy of ERT

针对交叉反应性免疫物质阴性的经典型婴儿庞贝病患者,制定早期诊断和治疗算法:提高酶替代疗法疗效的一步。

Banugaria, Suhrad G; Prater, Sean N; Patel, Trusha T; Dearmey, Stephanie M; Milleson, Christie; Sheets, Kathryn B; Bali, Deeksha S; Rehder, Catherine W; Raiman, Julian A J; Wang, Raymond A; Labarthe, Francois; Charrow, Joel; Harmatz, Paul; Chakraborty, Pranesh; Rosenberg, Amy S; Kishnani, Priya S

Skeletal muscle pathology of infantile Pompe disease during long-term enzyme replacement therapy

婴儿型庞贝病长期酶替代疗法期间的骨骼肌病理

Prater, Sean N; Patel, Trusha T; Buckley, Anne F; Mandel, Hanna; Vlodavski, Eugene; Banugaria, Suhrad G; Feeney, Erin J; Raben, Nina; Kishnani, Priya S

The emerging phenotype of long-term survivors with infantile Pompe disease

婴儿型庞贝病长期生存者的新兴表型

Prater, Sean N; Banugaria, Suhrad G; DeArmey, Stephanie M; Botha, Eleanor G; Stege, Erin M; Case, Laura E; Jones, Harrison N; Phornphutkul, Chanika; Wang, Raymond Y; Young, Sarah P; Kishnani, Priya S

Predicting cross-reactive immunological material (CRIM) status in Pompe disease using GAA mutations: lessons learned from 10 years of clinical laboratory testing experience

利用GAA基因突变预测庞贝病交叉反应性免疫物质(CRIM)状态:10年临床实验室检测经验的启示

Bali, Deeksha S; Goldstein, Jennifer L; Banugaria, Suhrad; Dai, Jian; Mackey, Joanne; Rehder, Catherine; Kishnani, Priya S

Persistence of high sustained antibodies to enzyme replacement therapy despite extensive immunomodulatory therapy in an infant with Pompe disease: need for agents to target antibody-secreting plasma cells

一名患有庞贝病的婴儿尽管接受了广泛的免疫调节治疗,但酶替代疗法仍持续存在高水平的抗体:需要靶向抗体分泌浆细胞的药物

Banugaria, Suhrad G; Patel, Trusha T; Mackey, Joanne; Das, Stuti; Amalfitano, Andrea; Rosenberg, Amy S; Charrow, Joel; Chen, Y-T; Kishnani, Priya S