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

Final Results of the ILLUMINATE-A Phase 3 Clinical Trial of Lumasiran for Primary Hyperoxaluria 1

Lumasiran治疗原发性高草酸尿症的ILLUMINATE-A 3期临床试验最终结果1

Frishberg, Yaacov; Saland, Jeffrey M; Lieske, John C; Du, Weiming; Coenen, Martin; Hogan, Julien; Sellier-Leclerc, Anne-Laure; Groothoff, Jaap W; Kaspar, Cristin; Gansner, John M; Hulton, Sally-Anne

Transition of patients with hereditary nephropathies from paediatric to adult care

遗传性肾病患者从儿科过渡到成人护理

Perez Gomez, Maria Vanessa; Costea, George-Claudiu; Claus, Laura; Cornec-Le Gall, Emilie; van Eerde, Albertien M; Lemoine, Sandrine; Groothoff, Jaap; Levtchenko, Elena; Klein, Luisa; Pape, Lars; Müller, Roman-Ulrich; Liebau, Max C

New opportunities for accessing promising non-oncological orphan drugs

获取有前景的非肿瘤孤儿药的新机遇

Hollak, Carla E M; Reparon-Schuijt, Carelle C; Verdeyen, Kathelijn; Deesker, Lisa J; Groothoff, Jaap W; Vos, Josephine M I; Huisman, Atse H; van der Flier, Silvia; Pisters-van Roy, Anke A M G

PHYOX3: Nedosiran Long-Term Safety and Efficacy in Patients With Primary Hyperoxaluria Type 1

PHYOX3:奈多西兰治疗原发性高草酸尿症1型患者的长期安全性和有效性

Lieske, John C; Ariceta, Gema; Groothoff, Jaap W; Lipkin, Graham; Moochhala, Shabbir H; Schalk, Gesa; Sellier-Leclerc, Anne-Laure; Torres, Sara Estupiñan; Rawson, Verity; Zhou, Jing; Hoppe, Bernd

Controlled access to lumasiran in primary hyperoxaluria type 1: evaluation of a new access route for orphan drugs in the Netherlands

在荷兰,对一种新的孤儿药获取途径进行评估,以控制原发性高草酸尿症1型患者获得鲁马西兰的途径。

Deesker, Lisa J; Franssen, Casper F M; Dorresteijn, Eiske; van de Kar, Nicole C A J; Nurmohamed, S Azam; Severs, David; Garrelfs, Sander F; Pisters-van Roy, Anke A M G; Hollak, Carla E M; Groothoff, Jaap W

Adult outcomes of childhood kidney replacement therapy in Europe from 2008 to 2019: an ERA Registry study

2008年至2019年欧洲儿童肾脏替代疗法对成年患者结局的影响:一项ERA注册研究

Montez de Sousa, Iris R; Bonthuis, Marjolein; Kramer, Anneke; Ordoñez, Flor Angel; de la Cerda Ojeda, Francisco; Rydell, Helena; Helve, Jaakko; Groothoff, Jaap W; Hommel, Kristine; Buchwinkler, Lukas; Segelmark, Mårten; Arici, Mustafa; Palsson, Runolfur; Bell, Samira; Trujillo-Alemán, Sara; Bakkaloglu, Sevcan A; Sørensen, Søren S; Vila, Anna; Ortiz, Alberto; Stel, Vianda S; Jager, Kitty J

Global access to management of primary hyperoxaluria: a survey on behalf of OxalEurope, G&K Working Group of the ERA and ESPN

全球原发性高草酸尿症治疗可及性:一项由 OxalEurope、ERA G&K 工作组和 ESPN 联合开展的调查

Deesker, Lisa J; Oubram, Laila; Almardini, Reham; Baum, Michelle A; Bonilla-Felix, M; Figueres, Lucile; Garrelfs, Sander F; Groothoff, Jaap W; Ekulu, Pépé M; Müller, Roman-Ulrich; Oosterveld, Michiel J S; Qian, Shen; Sayer, John A; Soliman, Neveen; Moochhala, Shabbir H; Bacchetta, Justine

Human glyoxylate metabolism revisited: New insights pointing to multi-organ involvement with implications for siRNA-based therapies in primary hyperoxaluria

重新审视人类乙醛酸代谢:新见解揭示多器官参与及其对原发性高草酸尿症siRNA疗法的意义

Wanders, Ronald J A; Groothoff, Jaap W; Deesker, Lisa J; Salido, Eduardo; Garrelfs, Sander F

Natural History of Advanced Primary Hyperoxaluria Type 1: A Retrospective Study

晚期原发性高草酸尿症1型自然史:一项回顾性研究

Lieske, John C; Groothoff, Jaap W; Frishberg, Yaacov; Garrelfs, Sander F; Milliner, Dawn S; Magen, Daniella; Sellier-Leclerc, Anne-Laure; Shasha-Lavsky, Hadas; Bakkaloǧlu, Sevcan; Lemoine, Sandrine; Fuster, Daniel G; Devresse, Arnaud; Cytter-Kuint, Ruth; Kajbaf, Farshad; Du, Weiming; Gansner, John M

Anterior Cruciate Ligament Reconstruction With Bone-Patellar Tendon-Bone Autograft With Concomitant Meniscal Allograft Transplantation

采用自体骨-髌腱-骨移植进行前交叉韧带重建,同时进行半月板同种异体移植

Groothoff, Jonathan D; Villa, Richard S; Glover, Mark A; Mason, Thomas W; Fiegen, Anthony P; van der List, Jelle P; Waterman, Brian R