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

The global impact of imiglucerase therapy in children with Gaucher disease types 1 and 3: a real-world analysis from the International Collaborative Gaucher Group Gaucher Registry

伊米苷酶疗法对1型和3型戈谢病患儿的全球影响:来自国际戈谢病协作组戈谢病注册中心的真实世界分析

Mistry, Pramod K; Carwile, Jenny L; Burrow, Thomas Andrew; Tantawy, Azza Abdel Gawad; Camelo, Jose Simon; Ganesh, Jaya; Batsu, Isabela; Perichon, Maria Gabriela; Oliveira-Dos-Santos, Antonio; Batista, Julie L

Gaucher disease type 3 from infancy through adulthood: a conceptual model of signs, symptoms, and impacts associated with ataxia and cognitive impairment

从婴儿期到成年期的戈谢病3型:共济失调和认知障碍相关体征、症状和影响的概念模型

Schiffmann, Raphael; Turnbull, James; Krupnick, Robert; Pulikottil-Jacob, Ruth; Gwaltney, Chad; Hamed, Alaa; Batsu, Isabela; Heine, Walter; Mengel, Eugen

Qualitative Study of the Patient Experience with Venglustat for Gaucher Disease Type 3 in a Phase 2 Open-Label, Multicenter, Multinational Study (LEAP)

一项针对戈谢病 3 型患者使用 Venglustat 的 2 期开放标签、多中心、多国研究 (LEAP) 的定性研究

Schiffmann, Raphael; Mengel, Eugen; Wallace, Mary; Rochmann, Camille; Turnbull, James; Krupnick, Robert; Gwaltney, Chad; Pulikottil-Jacob, Ruth; Batsu, Isabela; Zheng, Riliang; Hamed, Alaa

The diagnostic journey for patients with late-onset GM2 Gangliosidoses

晚发性GM2神经节苷脂病患者的诊断历程

Lopshire, Mariah C; Tifft, Cynthia; Burns, John; Gould, Rebecca; Zheng, Riliang; Batsu, Isabela

Treatment adherence and effect of concurrent statin intensity on the efficacy and safety of alirocumab in a real-life setting: results from ODYSSEY APPRISE

在真实世界环境中,治疗依从性以及同时使用他汀类药物强度对阿利西尤单抗疗效和安全性的影响:ODYSSEY APPRISE 研究结果

Banach, Maciej; López-Sendon, José Luis; Averna, Maurizio; Cariou, Bertrand; Loy, Megan; Manvelian, Garen; Batsu, Isabela; Poulouin, Yann; Gaudet, Daniel