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

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

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

Summary of Research: Risdiplam Treatment Following Onasemnogene Abeparvovec in Individuals with Spinal Muscular Atrophy: A Multicenter Case Series

研究总结:脊髓性肌萎缩症患者在接受奥那西姆诺基因阿贝帕沃韦治疗后接受利司迪普拉姆治疗:一项多中心病例系列研究

Svoboda, Melissa D; Kuntz, Nancy; Leon-Astudillo, Carmen; Byrne, Barry J; Krueger, Jena; Kwon, Jennifer M; Sieburg, Cory; Castro, Diana

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

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

Current clinical applications of AAV-mediated gene therapy

目前AAV介导的基因治疗的临床应用

Byrne, Barry J; Flanigan, Kevin M; Matesanz, Susan E; Finkel, Richard S; Waldrop, Megan A; D'Ambrosio, Eleonora S; Johnson, Nicholas E; Smith, Barbara K; Bönnemann, Carsten; Carrig, Sean; Rossano, Joseph W; Greenberg, Barry; Lalaguna, Laura; Lara-Pezzi, Enrique; Subramony, Sub; Corti, Manuela; Mercado-Rodriguez, Claudia; Leon-Astudillo, Carmen; Ahrens-Nicklas, Rebecca; Bharucha-Goebel, Diana; Gao, Guangping; Gessler, Dominic J; Hwu, Wuh-Liang; Chien, Yin-Hsiu; Lee, Ni-Chung; Boye, Sanford L; Boye, Shannon E; George, Lindsey A

Differences in swallowing efficacy of disease modifying treatment between infants receiving pre-symptomatic and symptomatic administration

接受疾病改善治疗的婴儿在症状前和症状后给药方面,吞咽疗效存在差异

McGrattan, Katlyn Elizabeth; Mohr, Alicia Hofelich; Miles, Anna; Allen, Jacqui; Ochura, Juliet; Hernandez, Kayla; Walsh, Katie; Rao, Vamshi; Stevens, Melanie; McGhee, Heather; Nichols, Keeley; Turksi, Morgan Elaine; Spoden, Abigail; Wilson, Irena; Coker, Mackenzi; Leon-Astudillo, Carmen; Smith, Leann Schow; Brandsema, John; Farah, Hiba; Welc, Julia; Levy, Deborah; Clements, Miranda; Duong, Tina; Young, Sally Dunaway; Schenck, Graham; Richardson, Randal; Karachunski, Peter; Brown, Ashley; Brown, Allison; Castro, Diana; Darras, Basil T; Graham, Robert J

Risdiplam treatment following onasemnogene abeparvovec in individuals with spinal muscular atrophy: a multicenter case series

在脊髓性肌萎缩症患者中,接受奥纳西姆诺基因阿贝帕沃韦治疗后,利司他普拉姆治疗:一项多中心病例系列研究

Svoboda, Melissa D; Kuntz, Nancy; Leon-Astudillo, Carmen; Byrne, Barry J; Krueger, Jena; Kwon, Jennifer M; Sieburg, Cory; Castro, Diana

Thrombotic microangiopathy following systemic AAV administration is dependent on anti-capsid antibodies

全身性AAV给药后发生的血栓性微血管病依赖于抗衣壳抗体。

Salabarria, Stephanie M; Corti, Manuela; Coleman, Kirsten E; Wichman, Megan B; Berthy, Julie A; D'Souza, Precilla; Tifft, Cynthia J; Herzog, Roland W; Elder, Melissa E; Shoemaker, Lawrence R; Leon-Astudillo, Carmen; Tavakkoli, Fatemeh; Kirn, David H; Schwartz, Jonathan D; Byrne, Barry J

Twelve Tips to Succeed as Health Profession Clinical Educator in Resource-Limited Settings

在资源匮乏的环境中,成为一名成功的卫生专业临床教育工作者的十二条建议

Alismail, Abdullah; Esteitie, Rania; Leon-Astudillo, Carmen; Pantaleón García, Jezreel; Sangli, Swathi; Kumar Sunil, Sriram

Current avenues of gene therapy in Pompe disease

庞贝病基因治疗的最新进展

Leon-Astudillo, Carmen; Trivedi, Prasad D; Sun, Ramon C; Gentry, Matthew S; Fuller, David D; Byrne, Barry J; Corti, Manuela

Secondary hemophagocytic lymphohistiocytosis following Zolgensma therapy: An evolving story on the innate response to systemic gene therapy

Zolgensma治疗后继发性噬血细胞性淋巴组织细胞增生症:系统性基因治疗先天反应的演变历程

Byrne, Barry J; Elder, Melissa; Leon-Astudillo, Carmen; Corti, Manuela