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

A TMPRSS6-inhibiting mAb improves disease in a β-thalassemia mouse model and reduces iron in healthy humans

TMPRSS6抑制剂单克隆抗体可改善β-地中海贫血小鼠模型中的疾病症状,并降低健康人体内铁含量。

Lob, Heinrich E; Singh, Nikhil; Mohammadi, Kusha; Ivanova, Larisa; Crowell, Beth; Kim, Hyon J; Kravets, Leah; Das, Nanditha M; Ray, Yonaton; Kim, Jee Hae; Rottey, Sylvie; Labriola-Tompkins, Emily; Hassan, Hazem E; Farrelly, Lorna; Chin, Harvey F; Preda, Marilena; Spencer Noakes, Leigh; Saotome, Kei; Franklin, Matthew; Retter, Marc W; Karayusuf, Elif; Flanagan, John J; Olson, William; Nannuru, Kalyan C; Idone, Vincent; Burczynski, Michael E; Harari, Olivier A; Perlee, Lorah; Van Lancker, Griet; Murphy, Andrew J; Economides, Aris N; Hatsell, Sarah J

The patient experience of CHAPLE disease: results from interviews conducted as part of a clinical trial for an ultra-rare condition

CHAPLE 病患者体验:一项针对罕见病临床试验的访谈结果

Litcher-Kelly, Leighann; Ozen, Ahmet; Ollis, Sarah; Feldman, Hagit Baris; Yaworsky, Andrew; Medrano, Paolo; Chongsrisawat, Voranush; Perlee, Lorah; Walker, Marisa; Pradeep, Sharanya; Turner-Bowker, Diane M; Kurolap, Alina; Adiv, Orly Eshach; Lenardo, Michael J; Harari, Olivier A; Jalbert, Jessica J

Safety, Efficacy, and Patient-Reported Outcomes From a Phase 2 Randomized Trial of Pozelimab and Cemdisiran Combination in Patients With Paroxysmal Nocturnal Hemoglobinuria

一项评估波泽利单抗和西美沙酮联合治疗阵发性睡眠性血红蛋白尿患者的 II 期随机试验的安全性、有效性和患者报告结局

Jang, Jun-Ho; Wong, Raymond Siu Ming; Hartford, Christopher; Pavani, Rodrigo; Aurand, Lisa; Nguyen, Quang; Mohan, Kosalai; Meagher, Karoline; Sherman, Steven; Rofail, Diana; Perlee, Lorah; Souttou, Amal; Kelly, Richard J

Evaluating the efficacy and safety of pozelimab in patients with CD55 deficiency with hyperactivation of complement, angiopathic thrombosis, and protein-losing enteropathy disease: an open-label phase 2 and 3 study

评估 pozelimab 在伴有补体过度激活、血管病性血栓形成和蛋白丢失性肠病的 CD55 缺陷患者中的疗效和安全性:一项开放标签的 2 期和 3 期研究

Ozen, Ahmet; Chongsrisawat, Voranush; Sefer, Asena Pinar; Kolukisa, Burcu; Jalbert, Jessica J; Meagher, Karoline A; Brackin, Taylor; Feldman, Hagit Baris; Baris, Safa; Karakoc-Aydiner, Elif; Ergelen, Rabia; Fuss, Ivan J; Moorman, Heather; Suratannon, Narissara; Suphapeetiporn, Kanya; Perlee, Lorah; Harari, Olivier A; Yancopoulos, George D; Lenardo, Michael J

Pozelimab for CHAPLE disease: results from in-trial interviews and clinical outcome assessments

Pozelimab治疗CHAPLE疾病:试验中访谈和临床结果评估的结果

Litcher-Kelly, Leighann; Ozen, Ahmet; Ollis, Sarah; Feldman, Hagit Baris; Yaworsky, Andrew; Medrano, Paolo; Chongsrisawa, Voranush; Brackin, Taylor; Perlee, Lorah; Walker, Marisa; Pradeep, Sharanya; Lenardo, Michael J; Harari, Olivier A; Jalbert, Jessica J

Intravitreal Nesvacumab (Anti-Angiopoietin-2) Plus Aflibercept in Neovascular AMD: Phase 2 ONYX Randomized Trial

玻璃体内注射奈斯伐单抗(抗血管生成素-2)联合阿柏西普治疗新生血管性年龄相关性黄斑变性:ONYX 2期随机试验

Heier, Jeffrey S; Ho, Allen C; Boyer, David S; Csaky, Karl; Vitti, Robert; Perlee, Lorah; Chu, Karen W; Asmus, Friedrich; Leal, Sergio; Zeitz, Oliver; Cheng, Yenchieh; Schmelter, Thomas; Brown, David M

Cat allergen exposure in a naturalistic exposure chamber: A prospective observational study in cat-allergic subjects

在模拟自然环境的暴露舱中对猫过敏原进行暴露:一项针对猫过敏受试者的前瞻性观察研究

Yang, William H; Kelly, Suzanne; Haya, Laura; Mehri, Rym; Ramesh, Divya; DeVeaux, Michelle; Wang, Claire Q; Meier, Pretty; Narula, Sumit; Shawki, Furat; Pennington, Ryan; Perlee, Lorah; O'Brien, Meagan P

INTRAVITREAL NESVACUMAB (ANTIANGIOPOIETIN 2) PLUS AFLIBERCEPT IN DIABETIC MACULAR EDEMA: Phase 2 RUBY Randomized Trial

玻璃体内注射奈伐单抗(抗血管生成素 2)联合阿柏西普治疗糖尿病性黄斑水肿:2 期 RUBY 随机试验

Brown, David M; Boyer, David S; Csaky, Karl; Vitti, Robert; Perlee, Lorah; Chu, Karen W; Asmus, Friedrich; Leal, Sergio; Zeitz, Oliver; Cheng, Yenchieh; Schmelter, Thomas; Heier, Jeffrey S

Clinical validation of a genetic model to estimate the risk of developing choroidal neovascular age-related macular degeneration

临床验证用于评估脉络膜新生血管性年龄相关性黄斑变性风险的遗传模型

Hageman, Gregory S; Gehrs, Karen; Lejnine, Serguei; Bansal, Aruna T; Deangelis, Margaret M; Guymer, Robyn H; Baird, Paul N; Allikmets, Rando; Deciu, Cosmin; Oeth, Paul; Perlee, Lorah T

Identification of diagnostic biomarkers for infection in premature neonates.

早产儿感染诊断生物标志物的鉴定

Kingsmore Stephen F, Kennedy Neil, Halliday Henry L, Van Velkinburgh Jennifer C, Zhong Shengiang, Gabriel Vanessa, Grant Judith, Beavis William D, Tchernev Velizar T, Perlee Lorah, Lejnine Serguei, Grimwade Brian, Sorette Martin, Edgar J David M