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

Lucerastat, an oral therapy for Fabry disease: results from a pivotal randomized phase 3 study and its open-label extension

Lucerastat,一种用于治疗法布里病的口服药物:一项关键性随机3期研究及其开放标签扩展研究的结果

Nordbeck, Peter; Goker-Alpan, Ozlem; Bernat, John A; Germain, Dominique P; Giraldo, Pilar; Jovanovic, Ana; Kimonis, Virginia; Nicholls, Kathleen; Rockman-Greenberg, Cheryl; Schiffmann, Raphael; Thomas, Mark; Tylki-Szymanska, Anna; Wallace, Eric; Welford, Richard W D; West, Michael L; Clozel, Martine; Frey, Aline; Trokan, Luba; Mueller, Markus S; Vogler, Markus; Wanner, Christoph; Hughes, Derralynn

Disorder-Engineered Hybrid Plasmonic Cavities for Emission Control of Defects in hBN

利用无序工程化混合等离子体腔控制六方氮化硼缺陷的发射

Genc, Sinan; Yücel, Oǧuzhan; Aǧlarcı, Furkan; Rodriguez-Fernandez, Carlos; Yilmaz, Alpay; Caglayan, Humeyra; Ateş, Serkan; Bek, Alpan

2025 Consensus Clinical Management Guidelines for Niemann-Pick Disease Type C

2025年尼曼-匹克病C型共识临床管理指南

Hiwot, Tarekegn; Porter, Forbes D; Bremova-Ertl, Tatiana; Ramaswami, Uma; Hastings, Caroline; Héron, Bénédicte; Hopkin, Justin; Melville, Joella; Amartino, Hernan; Del Toro, Mireia; Deodato, Federica; Ezgü, Fatih; Gissen, Paul; Gibson, James B; Ficicioglu, Can; Giugliani, Roberto; Staretz-Chacham, Orna; Platt, Frances; Guffon, Nathalie; Julich, Kristina; Kresojević, Nikola; Lehman, Anna; Nadjar, Yann; Schneider, Susanne A; Jones, Simon; Mengel, Eugen; Tchan, Michel; Walterfang, Mark; Goker-Alpan, Ozlem; Dawson, Charlotte; Cowie, Sandra; Mathieson, Toni; Berry-Kravis, Elizabeth; Patterson, Marc C

Maternal and Fetal Outcomes in Imiglucerase-Treated Patients With Gaucher Disease: Real-World Evidence From the International Collaborative Gaucher Group (ICGG) Gaucher Registry Pregnancy Sub-Registry

伊米苷酶治疗戈谢病患者的母婴结局:来自国际戈谢病协作组(ICGG)戈谢病登记妊娠子登记的真实世界证据

Revel-Vilk, Shoshana; Deegan, Patrick; Day-Salvatore, Debra; Goker-Alpan, Ozlem; Mengel, Eugen; Batista, Julie L; Carwile, Jenny L; Perichon, Gabriela; Ficicioglu, Can

Improvement of Bone Mineral Density in Patients with Type 1 Gaucher Disease Treated with Velaglucerase Alfa: Results from Clinical Studies

使用维拉葡糖苷酶α治疗1型戈谢病患者可改善骨矿物质密度:临床研究结果

Zimran, Ari; Botha, Jaco; Eastell, Richard; Ficicioglu, Can; Finkelman, Richard D; Frydman, Dafna; Giraldo, Pilar; Goker-Alpan, Ozlem; Kishnani, Priya S; Lau, Heather; Ruhrman-Shahar, Noa; Hughes, Derralynn A

Safety of home administration of cipaglucosidase alfa plus miglustat in late-onset Pompe disease: results from multiple clinical trials

居家使用西帕葡萄糖苷酶α联合米格鲁司他治疗晚发型庞贝病的安全性:多项临床试验的结果

Andersen, Henning; Díaz-Manera, Jordi; Goker-Alpan, Ozlem; Mozaffar, Tahseen; Sitaraman Das, Sheela; Fox, Brian; Amon, Farah; O'Brien-Prince, Kinesha; Goldman, Mitchell; Holdbrook, Fred; Jain, Vipul; Byrne, Barry J

Dupilumab opens a therapeutic window in food protein-induced enterocolitis syndrome by unlicensing dendritic cells

Dupilumab 通过激活树突状细胞,为食物蛋白诱导的肠炎综合征开辟了治疗窗口。

Plassmeyer, Matthew; Enav, Naomi; Girgis, Michael; Paige, Mikell; Todd, Linda; Israni, Laureana; Alpan, Oral

Safety and Efficacy of Intravenous Immune Globulin 10% (BIVIGAM(®)) in Children with Primary Immune Deficiency

静脉注射10%免疫球蛋白(BIVIGAM®)治疗原发性免疫缺陷儿童的安全性和有效性

Melamed, Isaac; Walter, Jolan E; Alpan, Oral; Leiding, Jennifer W

Sex Differences in Circulating Inflammatory, Immune, and Tissue Growth Markers Associated with Fabry Disease-Related Cardiomyopathy

法布里病相关心肌病中循环炎症、免疫和组织生长标志物的性别差异

Ivanova, Margarita M; Dao, Julia; Friedman, Andrew; Kasaci, Neil; Goker-Alpan, Ozlem

Efficacy and safety of avalglucosidase alfa in patients with late-onset Pompe disease after 145 weeks of treatment during the COMET trial

COMET试验中,经过145周的治疗,评估了阿伐葡萄糖苷酶α对晚发型庞贝病患者的疗效和安全性。

Kishnani, Priya S; Díaz-Manera, Jordi; Illarioshkin, Sergey; van der Ploeg, Ans T; Clemens, Paula R; Day, John W; Toscano, Antonio; Kushlaf, Hani; Ladha, Shafeeq; Attarian, Shahram; Carvalho, Gerson; Kostera-Pruszczyk, Anna; Erdem-Özdamar, Sevim; Goker-Alpan, Ozlem; Mozaffar, Tahseen; Straub, Volker; Roberts, Mark; Haack, Kristina An; Huynh-Ba, Olivier; Tammireddy, Swathi; Periquet, Magali; Thibault, Nathan; Zhou, Tianyue; Dimachkie, Mazen M; Schoser, Benedikt