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

Intrathecal onasemnogene abeparvovec for treatment-experienced patients with spinal muscular atrophy: a phase 3b, open-label trial

鞘内注射onasemnogene abeparvovec治疗既往接受过治疗的脊髓性肌萎缩症患者：一项3b期开放标签试验

期刊:Nature Medicine
影响因子:50
doi:10.1038/s41591-025-04119-2
Kwon, Jennifer M; Munell, Francina; Le Goff, Laure; Yuge, Kotaro; Kato, Tamaki; Cances, Claude; De Waele, Liesbeth; Woodcock, Ian R; Mercuri, Eugenio M; Proud, Crystal M; Darras, Basil T; Hayes, Leslie H; Oskoui, Maryam; Visootsak, Jeannie; Williams, Gemma; Ilić, Andreja; Yang, Lina; van der Pol, W Ludo
神经科学
肌萎缩症
发表日期：2026
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Author Correction: Intrathecal onasemnogene abeparvovec for treatment-experienced patients with spinal muscular atrophy: a phase 3b, open-label trial

作者更正：鞘内注射onasemnogene abeparvovec治疗既往接受过治疗的脊髓性肌萎缩症患者：一项3b期开放标签试验

期刊:Nature Medicine
影响因子:50
doi:10.1038/s41591-026-04233-9
Kwon, Jennifer M; Munell, Francina; Le Goff, Laure; Yuge, Kotaro; Kato, Tamaki; Cances, Claude; De Waele, Liesbeth; Woodcock, Ian R; Mercuri, Eugenio M; Proud, Crystal M; Darras, Basil T; Hayes, Leslie H; Oskoui, Maryam; Visootsak, Jeannie; Williams, Gemma; Ilić, Andreja; Yang, Lina; van der Pol, W Ludo
神经科学
肌萎缩症
发表日期：2026
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Intrathecal onasemnogene abeparvovec in treatment-naive patients with spinal muscular atrophy: a phase 3, randomized controlled trial

鞘内注射onasemnogene abeparvovec治疗初治脊髓性肌萎缩症患者：一项3期随机对照试验

期刊:Nature Medicine
影响因子:50
doi:10.1038/s41591-025-04103-w
Proud, Crystal M; Vũ, Dũng Chí; Wilmshurst, Jo M; Sanmaneechai, Oranee; Gulati, Sheffali; Xiong, Hui; Moreno, Hugo C; Tay, Stacey Kiat Hong; Thong, Meow-Keong; Born, Alfred Peter; Banzzatto Ortega, Adriana; Jong, Yuh-Jyh; Al-Muhaizea, Mohammad A; Lee, Anna W; Visootsak, Jeannie; Tauscher-Wisniewski, Sitra; Alecu, Iulian; Parlikar, Rutvick; Finkel, Richard S
神经科学
肌萎缩症
发表日期：2026
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Two-Year Outcomes Following Delandistrogene Moxeparvovec Treatment in Ambulatory Patients with Duchenne Muscular Dystrophy: Phase 3 EMBARK Trial

Delandistrogene Moxeparvovec 治疗可活动杜氏肌营养不良症患者的两年疗效：3 期 EMBARK 试验

期刊:Neurology and Therapy
影响因子:4.8
doi:10.1007/s40120-025-00879-8
Mendell, Jerry R; Muntoni, Francesco; McDonald, Craig M; Mercuri, Eugenio M; Ciafaloni, Emma; Komaki, Hirofumi; Leon-Astudillo, Carmen; Nascimento, Andrés; Proud, Crystal; Schara-Schmidt, Ulrike; Veerapandiyan, Aravindhan; Zaidman, Craig M; Furgerson, Matthew; Ding, Kai; Singh, Preeti; Potter, Rachael; Asher, Damon R; Murphy, Alexander P; Reid, Carol; Hooper, Gregory; Torre, Carmen O; Manfrini, Marianna; Rodino-Klapac, Louise R
发表日期：2026
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AAV gene therapy for Duchenne muscular dystrophy: the EMBARK phase 3 randomized trial.

AAV基因疗法治疗杜氏肌营养不良症：EMBARK 3期随机试验

期刊:Nature Medicine
影响因子:50
doi:10.1038/s41591-024-03304-z
Mendell Jerry R, Muntoni Francesco, McDonald Craig M, Mercuri Eugenio M, Ciafaloni Emma, Komaki Hirofumi, Leon-Astudillo Carmen, Nascimento Andrés, Proud Crystal, Schara-Schmidt Ulrike, Veerapandiyan Aravindhan, Zaidman Craig M, Guridi Maitea, Murphy Alexander P, Reid Carol, Wandel Christoph, Asher Damon R, Darton Eddie, Mason Stefanie, Potter Rachael A, Singh Teji, Zhang Wenfei, Fontoura Paulo, Elkins Jacob S, Rodino-Klapac Louise R
其它
发表日期：2025
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Open-label phase IV trial evaluating nusinersen after onasemnogene abeparvovec in children with spinal muscular atrophy

一项开放标签的IV期试验评估了在脊髓性肌萎缩症患儿中，接受onasemnogene abeparvovec治疗后使用nusinersen的疗效。

期刊:Journal of Clinical Investigation
影响因子:13.6
doi:10.1172/JCI193956
Proud, Crystal M; Finkel, Richard S; Parsons, Julie A; Masson, Riccardo; Brandsema, John F; Kuntz, Nancy L; Foster, Richard; Li, Wenjing; Littauer, Ross; Sohn, Jihee; Fradette, Stephanie; Youn, Bora; Paradis, Angela D
神经科学
肌萎缩症
发表日期：2025
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Spinal Muscular Atrophy Update in Best Practices: Recommendations for Treatment Considerations

脊髓性肌萎缩症最佳实践更新：治疗注意事项建议

期刊:Neurology-Clinical Practice
影响因子:3.2
doi:10.1212/CPJ.0000000000200374
Schroth, Mary K; Deans, Jennifer; Bharucha Goebel, Diana X; Burnette, W Bryan; Darras, Basil T; Elsheikh, Bakri H; Felker, Marcia V; Klein, Andrea; Krueger, Jena; Proud, Crystal M; Veerapandiyan, Aravindhan; Graham, Robert J
神经科学
肌萎缩症
发表日期：2025
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Real-World Outcomes in Patients with Spinal Muscular Atrophy Treated with Onasemnogene Abeparvovec Monotherapy: Findings from the RESTORE Registry

使用 Onasemnogene Abeparvovec 单药治疗脊髓性肌萎缩症患者的真实世界结果：来自 RESTORE 注册研究的发现

期刊:Journal of Neuromuscular Diseases
影响因子:3.4
doi:10.3233/JND-230122
Servais, Laurent; Day, John W; De Vivo, Darryl C; Kirschner, Janbernd; Mercuri, Eugenio; Muntoni, Francesco; Proud, Crystal M; Shieh, Perry B; Tizzano, Eduardo F; Quijano-Roy, Susana; Desguerre, Isabelle; Saito, Kayoko; Faulkner, Eric; Benguerba, Kamal M; Raju, Dheeraj; LaMarca, Nicole; Sun, Rui; Anderson, Frederick A; Finkel, Richard S
神经科学
REST
肌萎缩症
发表日期：2024
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Examining Real-World Adherence to Nusinersen for the Treatment of Spinal Muscular Atrophy Using Two Large US Data Sources

利用两个大型美国数据源考察诺西那生治疗脊髓性肌萎缩症的真实世界依从性

期刊:Advances in Therapy
影响因子:4
doi:10.1007/s12325-022-02414-9
Youn, Bora; Proud, Crystal M; Wang, Nasha; Hou, Qiang; Viscidi, Emma; Eaton, Susan; Paradis, Angela D; Neville, Bridget A; Johnson, Nicole B
神经科学
肌萎缩症
发表日期：2023
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Combination disease-modifying treatment in spinal muscular atrophy: A proposed classification

脊髓性肌萎缩症的联合疾病修饰治疗：一种分类方案

期刊:Annals of Clinical and Translational Neurology
影响因子:3.9
doi:10.1002/acn3.51889
Proud, Crystal M; Mercuri, Eugenio; Finkel, Richard S; Kirschner, Janbernd; De Vivo, Darryl C; Muntoni, Francesco; Saito, Kayoko; Tizzano, Eduardo F; Desguerre, Isabelle; Quijano-Roy, Susana; Benguerba, Kamal; Raju, Dheeraj; Faulkner, Eric; Servais, Laurent
神经科学
肌萎缩症
发表日期：2023
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