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

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

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

Nedosiran Safety and Efficacy in PH1: Interim Analysis of PHYOX3

Nedosiran 在 PH1 中的安全性和有效性:PHYOX3 的中期分析

Groothoff, Jaap; Sellier-Leclerc, Anne-Laure; Deesker, Lisa; Bacchetta, Justine; Schalk, Gesa; Tönshoff, Burkhard; Lipkin, Graham; Lemoine, Sandrine; Bowman, Thomas; Zhou, Jing; Hoppe, Bernd

Intrafamilial Disease Heterogeneity in Primary Hyperoxaluria Type 1

原发性高草酸尿症1型的家族内疾病异质性

Deesker, Lisa J; Karacoban, Hazal A; Metry, Elisabeth L; Garrelfs, Sander F; Bacchetta, Justine; Boyer, Olivia; Collard, Laure; Devresse, Arnaud; Hayes, Wesley; Hulton, Sally-Anne; Martin-Higueras, Cristina; Moochhala, Shabbir H; Neuhaus, Thomas J; Oh, Jun; Prikhodina, Larisa; Sikora, Przemyslaw; Oosterveld, Michiel J S; Groothoff, Jaap W; Mandrile, Giorgia; Beck, Bodo B

Application of four pricing models for orphan medicines: a case study for lumasiran

四种孤儿药定价模式的应用:以卢马西兰为例

Rosenberg, Noa; Manders, Evert; van den Berg, Sibren; Deesker, Lisa J; Garrelfs, Sander F; de Visser, Saco J; Groothoff, Jaap W; Hollak, Carla E M

Determinants of Kidney Failure in Primary Hyperoxaluria Type 1: Findings of the European Hyperoxaluria Consortium

原发性高草酸尿症 1 型肾衰竭的决定因素:欧洲高草酸尿症联盟的研究结果

Metry, Elisabeth L; Garrelfs, Sander F; Deesker, Lisa J; Acquaviva, Cecile; D'Ambrosio, Viola; Bacchetta, Justine; Beck, Bodo B; Cochat, Pierre; Collard, Laure; Hogan, Julien; Ferraro, Pietro Manuel; Franssen, Casper F M; Harambat, Jérôme; Hulton, Sally-Anne; Lipkin, Graham W; Mandrile, Giorgia; Martin-Higueras, Cristina; Mohebbi, Nilufar; Moochhala, Shabbir H; Neuhaus, Thomas J; Prikhodina, Larisa; Salido, Eduardo; Topaloglu, Rezan; Oosterveld, Michiel J S; Groothoff, Jaap W; Peters-Sengers, Hessel

Genetic assessment in primary hyperoxaluria: why it matters

原发性高草酸尿症的基因评估:为什么它如此重要

Mandrile, Giorgia; Beck, Bodo; Acquaviva, Cecile; Rumsby, Gill; Deesker, Lisa; Garrelfs, Sander; Gupta, Asheeta; Bacchetta, Justine; Groothoff, Jaap

Improved Outcome of Infantile Oxalosis Over Time in Europe: Data From the OxalEurope Registry

欧洲婴儿草酸盐沉积症的治疗效果随时间推移而改善:来自 OxalEurope 注册中心的数据

Deesker, Lisa J; Garrelfs, Sander F; Mandrile, Giorgia; Oosterveld, Michiel J S; Cochat, Pierre; Deschênes, Georges; Harambat, Jérôme; Hulton, Sally-Anne; Gupta, Asheeta; Hoppe, Bernd; Beck, Bodo B; Collard, Laure; Topaloglu, Rezan; Prikhodina, Larisa; Salido, Eduardo; Neuhaus, Thomas; Groothoff, Jaap W; Bacchetta, Justine