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

Clinical modeling of motor function to predict treatment efficacy and enable in silico treatment comparisons in infantile-onset Pompe disease

利用运动功能临床模型预测治疗效果，并实现婴儿型庞贝病治疗方案的计算机模拟比较

期刊:Cpt-Pharmacometrics & Systems Pharmacology
影响因子:3
doi:10.1002/psp4.13287
Rachedi, Fatiha; Jreich, Rana; Sparks, Susan; Zaher, Atef; An Haack, Kristina; Granados, Alicia; Meng, Zhaoling
发表日期：2025
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The value of knowing: preferences for genetic testing to diagnose rare muscle diseases

了解的价值：对基因检测在诊断罕见肌肉疾病方面的偏好

期刊:Orphanet Journal of Rare Diseases
影响因子:3.5
doi:10.1186/s13023-024-03160-7
Mansfield, Carol; Boeri, Marco; Coulter, Josh; Baranowski, Eileen; Sparks, Susan; An Haack, Kristina; Hamed, Alaa
发表日期：2024
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Efficacy of avalglucosidase alfa on forced vital capacity percent predicted in treatment-naïve patients with late-onset Pompe disease: A pooled analysis of clinical trials

阿伐葡萄糖苷酶α对初治晚发型庞贝病患者用力肺活量占预计值的百分比的影响：一项临床试验汇总分析

期刊:Molecular Genetics and Metabolism Reports
影响因子:1.9
doi:10.1016/j.ymgmr.2024.101109
Mozaffar, Tahseen; Riou França, Lionel; Msihid, Jérôme; Shukla, Pragya; Proskorovsky, Irina; Zhou, Tianyue; Periquet, Magali; An Haack, Kristina; Pollissard, Laurence; Straub, Volker
发表日期：2024
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Efficacy and Safety of Avalglucosidase Alfa in Patients With Late-Onset Pompe Disease After 97 Weeks: A Phase 3 Randomized Clinical Trial

阿伐葡萄糖苷酶α治疗晚发型庞贝病患者97周后的疗效和安全性：一项3期随机临床试验

期刊:Jama Neurology
影响因子:21.3
doi:10.1001/jamaneurol.2023.0552
Kishnani, Priya S; Diaz-Manera, Jordi; Toscano, Antonio; Clemens, Paula R; Ladha, Shafeeq; Berger, Kenneth I; Kushlaf, Hani; Straub, Volker; Carvalho, Gerson; Mozaffar, Tahseen; Roberts, Mark; Attarian, Shahram; Chien, Yin-Hsiu; Choi, Young-Chul; Day, John W; Erdem-Ozdamar, Sevim; Illarioshkin, Sergey; Goker-Alpan, Ozlem; Kostera-Pruszczyk, Anna; van der Ploeg, Ans T; An Haack, Kristina; Huynh-Ba, Olivier; Tammireddy, Swathi; Thibault, Nathan; Zhou, Tianyue; Dimachkie, Mazen M; Schoser, Benedikt
发表日期：2023
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Measurement Properties of 2 Novel PROs, the Pompe Disease Symptom Scale and Pompe Disease Impact Scale, in the COMET Study

COMET 研究中两种新型患者报告结局指标（庞贝病症状量表和庞贝病影响量表）的测量特性

期刊:Neurology-Clinical Practice
影响因子:3.2
doi:10.1212/CPJ.0000000000200181
Dimachkie, Mazen M; Kishnani, Priya S; Ivanescu, Cristina; Flore, Giulio; Gwaltney, Chad; van der Beek, Nadine A M E; Hamed, Alaa; An Haack, Kristina; Pollissard, Laurence; Baranowski, Eileen; Sparks, Susan E; DasMahapatra, Pronabesh
MET
发表日期：2023
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The German CaRe high registry for familial hypercholesterolemia - Sex differences, treatment strategies, and target value attainment

德国 CaRe 高胆固醇血症登记研究——性别差异、治疗策略和目标值达成情况

期刊:Atherosclerosis Plus
影响因子:2.1
doi:10.1016/j.athplu.2023.06.001
März, Winfried; Schmidt, Nina; An Haack, Ira; Dressel, Alexander; Grammer, Tanja B; Kleber, Marcus E; Baessler, Andrea; Beil, F Ulrich; Gouni-Berthold, Ioanna; Julius, Ulrich; Kassner, Ursula; Katzmann, Julius L; Klose, Gerald; König, Christel; Koenig, Wolfgang; Koschker, Ann-Cathrin; Laufs, Ulrich; Merkel, Martin; Otte, Britta; Parhofer, Klaus G; Hengstenberg, Wibke; Schunkert, Heribert; Stach-Jablonski, Ksenija; Steinhagen-Thiessen, Elisabeth; Olivier, Christoph B; Hahmann, Harry; Krzossok, Stefan; Vogt, Anja; Müller-Wieland, Dirk; Schatz, Ulrike
代谢
发表日期：2023
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Qualitative interviews to improve patient-reported outcome measures in late-onset Pompe disease: the patient perspective

定性访谈在改善晚发型庞贝病患者报告结局指标方面的应用：患者视角

期刊:Orphanet Journal of Rare Diseases
影响因子:3.5
doi:10.1186/s13023-021-02067-x
Hamed, Alaa; An Haack, Kristina; Gwaltney, Chad; Baranowski, Eileen; Stewart, Andrew; Krupnick, Robert; Tyler, Margaret; Sparks, Susan; Paty, Jean
发表日期：2021
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Development and content validation of the Pediatric Oral Medicines Acceptability Questionnaires (P-OMAQ): patient-reported and caregiver-reported outcome measures

儿童口服药物接受度问卷（P-OMAQ）的开发和内容验证：患者自评和照护者自评结果指标

期刊:Journal of Patient-Reported Outcomes
影响因子:2.9
doi:10.1186/s41687-020-00246-1
Turner-Bowker, Diane M; An Haack, Kristina; Krohe, Meaghan; Yaworsky, Andrew; Vivas, Norma; Kelly, Masami; Chatterjee, Godhuli; Chaston, Emily; Mann, Erin; Reaney, Matthew
发表日期：2020
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How is the Pharmaceutical Industry Structured to Optimize Pediatric Drug Development? Existing Pediatric Structure Models and Proposed Recommendations for Structural Enhancement

制药行业的结构如何才能优化儿科药物的研发？现有儿科药物研发结构模型及结构改进建议

期刊:
影响因子:
doi:10.1007/s43441-020-00116-4
Severin, Thomas; Corriol-Rohou, Solange; Bucci-Rechtweg, Christina; An Haack, Kristina; Fuerst-Recktenwald, Sabine; Lepola, Pirkko; Norjavaara, Ensio; Dehlinger-Kremer, Martine; Haertter, Sebastian; Cheung, S Y Amy
发表日期：2020
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Clinical characteristics and genotypes in the ADVANCE baseline data set, a comprehensive cohort of US children and adolescents with Pompe disease

ADVANCE基线数据集（一项针对美国庞贝病患儿和青少年的综合队列研究）中的临床特征和基因型

期刊:Genetics in Medicine
影响因子:6.2
doi:10.1038/s41436-019-0527-9
Kishnani, Priya S; Gibson, James B; Gambello, Michael J; Hillman, Richard; Stockton, David W; Kronn, David; Leslie, Nancy D; Pena, Loren D M; Tanpaiboon, Pranoot; Day, John W; Wang, Raymond Y; Goldstein, Jennifer L; An Haack, Kristina; Sparks, Susan E; Zhao, Yang; Hahn, Si Houn
发表日期：2019
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