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

Intrathecal or intravenous AAV9-IDUA/RGX-111 at minimal effective dose prevents cardiac, skeletal and neurologic manifestations of murine MPS I.

鞘内或静脉注射最小有效剂量的 AAV9-IDUA/RGX-111 可预防小鼠 MPS I 的心脏、骨骼和神经系统症状

期刊:Molecular Therapy-Methods & Clinical Development
影响因子:4.7
doi:10.1016/j.omtm.2024.101369
Belur Lalitha R, Huber Avery K, Mantone Hillary, Robertson Mason, Smith Miles C, Karlen Andrea D, Kitto Kelley F, Ou Li, Whitley Chester B, Braunlin Elizabeth, Furcich Justin, Lund Troy C, Seelig Davis, Fairbanks Carolyn A, Buss Nicholas, Kim Kwi Hye, McIvor R Scott
神经科学
发表日期：2024
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Hurler Syndrome Glycosaminoglycans Decrease in Cerebrospinal Fluid without Brain-Targeted Therapy

赫勒氏综合征患者脑脊液中糖胺聚糖减少，且未接受脑靶向治疗

期刊:Annals of Neurology
影响因子:7.7
doi:10.1002/ana.26786
Lund, Troy C; Braunlin, Elizabeth; Polgreen, Lynda E; Gupta, Ashish O; Orchard, Paul J; Eisengart, Julie B
神经科学
发表日期：2023
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Prolonged Elevations of Factor VIII and von Willebrand Factor Antigen After Multisystem Inflammatory Syndrome in Children

儿童多系统炎症综合征后因子VIII和血管性血友病因子抗原持续升高

期刊:Journal of Pediatric Hematology Oncology
影响因子:0.8
doi:10.1097/MPH.0000000000002583
Boucher, Alexander A; Knutson, Stacie; Young, Luke; Evans, Michael D; Braunlin, Elizabeth; Zantek, Nicole D; Sharon, Bazak; Binstadt, Bryce A; Ryan, Meghan; Greene, Ryan; Mahmud, Shawn; Marmet, Jordan; Fischer, Gwenyth; Steiner, Marie E
炎症/感染
炎症
发表日期：2023
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Hematopoietic stem cell transplant for Hurler syndrome: does using bone marrow or umbilical cord blood make a difference?

造血干细胞移植治疗赫勒氏综合征：使用骨髓还是脐带血有区别？

期刊:Blood Advances
影响因子:7.1
doi:10.1182/bloodadvances.2022007212
Orchard, Paul J; Gupta, Ashish O; Eisengart, Julie B; Polgreen, Lynda E; Pollard, Laura M; Braunlin, Elizabeth; Pasquali, Marzia; Lund, Troy C
细胞生物学
发育与干细胞
干细胞
发表日期：2022
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Editorial: Cardiac issues in adults with mucopolysaccharidosis

社论：成人粘多糖贮积症的心脏问题

期刊:Frontiers in Cardiovascular Medicine
影响因子:2.9
doi:10.3389/fcvm.2022.1016386
Stepien, Karolina M; Braunlin, Elizabeth A
发表日期：2022
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Unmet Cardiac Clinical Needs in Adult Mucopolysaccharidoses

成人粘多糖贮积症患者未满足的心脏临床需求

期刊:Frontiers in Cardiovascular Medicine
影响因子:2.9
doi:10.3389/fcvm.2022.907175
Stepien, Karolina M; Braunlin, Elizabeth A
发表日期：2022
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Timing is everything: Clinical courses of Hunter syndrome associated with age at initiation of therapy in a sibling pair

时机至关重要：亨特综合征的临床病程与同胞兄弟姐妹开始治疗的年龄相关

期刊:Molecular Genetics and Metabolism Reports
影响因子:1.9
doi:10.1016/j.ymgmr.2022.100845
Grant, Nathan; Sohn, Young Bae; Ellinwood, N Matthew; Okenfuss, Ericka; Mendelsohn, Bryce A; Lynch, Leslie E; Braunlin, Elizabeth A; Harmatz, Paul R; Eisengart, Julie B
发表日期：2022
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The double shunt technique as a bridge to heart transplantation in a patient with pulmonary atresia with intact septum and right ventricular-dependent coronary circulation

双分流术作为肺动脉闭锁伴室间隔完整和右心室依赖性冠状动脉循环患者的心脏移植过渡治疗

期刊:
影响因子:
doi:10.1016/j.xjtc.2021.01.018
Said, Sameh M; Marey, Gamal; Greene, Ryan; Griselli, Massimo; Hiremath, Gurumurthy; Aggarwal, Varun; Braunlin, Elizabeth
发表日期：2021
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Recommendations for the management of MPS VI: systematic evidence- and consensus-based guidance

MPS VI 管理建议：基于系统证据和共识的指导

期刊:Orphanet Journal of Rare Diseases
影响因子:3.5
doi:10.1186/s13023-019-1080-y
Akyol, Mehmet Umut; Alden, Tord D; Amartino, Hernan; Ashworth, Jane; Belani, Kumar; Berger, Kenneth I; Borgo, Andrea; Braunlin, Elizabeth; Eto, Yoshikatsu; Gold, Jeffrey I; Jester, Andrea; Jones, Simon A; Karsli, Cengiz; Mackenzie, William; Marinho, Diane Ruschel; McFadyen, Andrew; McGill, Jim; Mitchell, John J; Muenzer, Joseph; Okuyama, Torayuki; Orchard, Paul J; Stevens, Bob; Thomas, Sophie; Walker, Robert; Wynn, Robert; Giugliani, Roberto; Harmatz, Paul; Hendriksz, Christian; Scarpa, Maurizio
发表日期：2019
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Recommendations for the management of MPS IVA: systematic evidence- and consensus-based guidance

MPS IVA 管理建议：基于系统证据和共识的指导

期刊:Orphanet Journal of Rare Diseases
影响因子:3.5
doi:10.1186/s13023-019-1074-9
Akyol, Mehmet Umut; Alden, Tord D; Amartino, Hernan; Ashworth, Jane; Belani, Kumar; Berger, Kenneth I; Borgo, Andrea; Braunlin, Elizabeth; Eto, Yoshikatsu; Gold, Jeffrey I; Jester, Andrea; Jones, Simon A; Karsli, Cengiz; Mackenzie, William; Marinho, Diane Ruschel; McFadyen, Andrew; McGill, Jim; Mitchell, John J; Muenzer, Joseph; Okuyama, Torayuki; Orchard, Paul J; Stevens, Bob; Thomas, Sophie; Walker, Robert; Wynn, Robert; Giugliani, Roberto; Harmatz, Paul; Hendriksz, Christian; Scarpa, Maurizio
发表日期：2019
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