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

Eladocagene Exuparvovec for the Treatment of Aromatic L-Amino Acid Decarboxylase Deficiency (AADCd): An Economic Evaluation from a US Perspective

埃拉多卡吉内·埃苏帕沃韦克治疗芳香族L-氨基酸脱羧酶缺乏症（AADCd）：美国视角下的经济评价

期刊:Pharmacoeconomics
影响因子:4.6
doi:10.1007/s40273-025-01542-8
Zhang, Rongrong; O'Connell, Thomas; Monteleone, Berrin; Teng, Yixi; Hwu, Paul Wuh-Liang; Castellano, Paul; Tomazos, Ioannis
发表日期：2026
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Clinically meaningful improvements after gene therapy for aromatic L-amino acid decarboxylase deficiency (AADCd) in the Peabody Developmental Motor Scale, Second Edition (PDMS-2) and correlation with Bayley-III scores and motor milestones

基因治疗后芳香族L-氨基酸脱羧酶缺乏症（AADCd）在皮博迪发育运动量表第二版（PDMS-2）中取得具有临床意义的改善，并与贝利婴幼儿发展量表第三版评分和运动里程碑相关

期刊:Orphanet Journal of Rare Diseases
影响因子:3.5
doi:10.1186/s13023-025-03584-9
Hwu, Wuh-Liang; Lee, Hui-Min; Peipert, John Devin; Zhang, Rongrong; Werner, Christian; Sierra, J Rafael; O'Connell, Thomas; Woolley, Jonathan J; Crowell, Marjorie; Wang, Antonia; Tomazos, Ioannis
发育与干细胞
基因治疗
多发性硬化症
发表日期：2025
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Intelligence quotient scores among early-treated phenylketonuria patients: results from a systematic literature review

早期治疗的苯丙酮尿症患者的智商评分：一项系统性文献综述的结果

期刊:Orphanet Journal of Rare Diseases
影响因子:3.5
doi:10.1186/s13023-025-03830-0
O'Sullivan, Fiona; Tomazos, Ioannis; van Spronsen, Francjan J; Szabo, Shelagh M; Venkataraman, Maanasa; Huria, Lavanya; Smith, Neil; Molony, Lachlan; Ingalls, Kim; Somera-Molina, Kathleen; Zhang, Rongrong; Harding, Cary O
发表日期：2025
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Estimating health state utilities for aromatic L-amino acid decarboxylase deficiency (AADCd) in the United States

估算美国芳香族L-氨基酸脱羧酶缺乏症（AADCd）的健康状态效用

期刊:Health and Quality of Life Outcomes
影响因子:3.4
doi:10.1186/s12955-025-02335-5
Monteleone, Berrin; Forster, Katie; Chua, Gin Nie; Zhang, Rongrong; Lloyd, Andrew; Castellano, Paul; Tomazos, Ioannis
发表日期：2025
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PLASMIC score to aid diagnosis of aHUS: an analysis of C5 inhibitor clinical trials and the PINC AI™ healthcare database

PLASMIC评分辅助诊断aHUS：C5抑制剂临床试验和PINC AI™医疗保健数据库分析

期刊:BMC Nephrology
影响因子:2.4
doi:10.1186/s12882-025-04156-6
Uriol-Rivera, Miguel G; Ernst, Frank R; Booth, John N 3rd; Comas, Àngels; Gasteyger, Christoph; Tomazos, Ioannis; Lum, Ching; Wang, Yan; Ávila, Ana I
发表日期：2025
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Predictors of cardiac disease in duchenne muscular dystrophy: a systematic review and evidence grading

杜氏肌营养不良症心脏疾病的预测因素：系统评价和证据分级

期刊:Orphanet Journal of Rare Diseases
影响因子:3.5
doi:10.1186/s13023-024-03372-x
Landfeldt, Erik; Alemán, Alberto; Abner, Sophia; Zhang, Rongrong; Werner, Christian; Tomazos, Ioannis; Lochmüller, Hanns; Quinlivan, Ros M; Wahbi, Karim
心脏病
发表日期：2024
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Predictors of Loss of Ambulation in Duchenne Muscular Dystrophy: A Systematic Review and Meta-Analysis

杜氏肌营养不良症患者丧失行走能力的预测因素：系统评价和荟萃分析

期刊:Journal of Neuromuscular Diseases
影响因子:3.4
doi:10.3233/JND-230220
Landfeldt, E; Alemán, A; Abner, S; Zhang, R; Werner, C; Tomazos, I; Ferizovic, N; Lochmüller, H; Kirschner, J
发表日期：2024
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Factors Associated with Respiratory Health and Function in Duchenne Muscular Dystrophy: A Systematic Review and Evidence Grading

杜氏肌营养不良症患者呼吸健康和功能相关因素：系统评价和证据分级

期刊:Journal of Neuromuscular Diseases
影响因子:3.4
doi:10.3233/JND-230094
Landfeldt, E; Aleman, A; Abner, S; Zhang, R; Werner, C; Tomazos, I; Lochmüller, H; Quinlivan, R M
发表日期：2024
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Predictors for improvement in patient-reported outcomes: post hoc analysis of a phase 3 randomized, open-label study of eculizumab and ravulizumab in complement inhibitor-naive patients with paroxysmal nocturnal hemoglobinuria

预测患者报告结局改善的因素：一项针对未接受补体抑制剂治疗的阵发性睡眠性血红蛋白尿患者的依库珠单抗和拉武珠单抗的3期随机、开放标签研究的事后分析

期刊:Annals of Hematology
影响因子:2.4
doi:10.1007/s00277-023-05483-0
Schrezenmeier, Hubert; Kulasekararaj, Austin; Mitchell, Lindsay; de Latour, Régis Peffault; Devos, Timothy; Okamoto, Shinichiro; Wells, Richard; Popoff, Evan; Cheung, Antoinette; Wang, Alice; Tomazos, Ioannis; Patel, Yogesh; Lee, Jong Wook
免疫/内分泌
发表日期：2024
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The burden of mitochondrial disease with associated seizures: systematic literature reviews of health-related quality of life, utilities, costs and healthcare resource use data

线粒体疾病合并癫痫的负担：健康相关生活质量、效用、成本和医疗资源利用数据的系统性文献综述

期刊:Orphanet Journal of Rare Diseases
影响因子:3.5
doi:10.1186/s13023-023-02945-6
Bertini, Enrico; Gregg, Emily; Bartlett, Chris; Senthilnathan, Vij; Arber, Mick; Watkins, Deborah; Graziadio, Sara; Tomazos, Ioannis
线粒体
癫痫
神经科学
发表日期：2023
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