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

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

Immunologic investigations into transgene directed immune-mediated myositis following delandistrogene moxeparvovec gene therapy

对接受 delandistrogene moxeparvovec 基因治疗后发生的转基因导向免疫介导性肌炎进行免疫学研究

Potter, Rachael A; Moeller, Ida H; Khan, Sohrab; Haegel, Hélène; Hollenstein, Andreas; Steiner, Guido; Wandel, Christoph; Murphy, Alexander P; Asher, Damon R; Palatinsky, Emanuel; Griffin, Danielle A; Mason, Stefanie; Iannaccone, Susan T; Zaidman, Craig M; Rodino-Klapac, Louise R

Evaluation of rAAVrh74 gene therapy vector seroprevalence by measurement of total binding antibodies in patients with Duchenne muscular dystrophy

通过测量杜氏肌营养不良症患者体内总结合抗体来评估rAAVrh74基因治疗载体的血清阳性率

Goedeker, Natalie L; Dharia, Sachi D; Griffin, Danielle A; Coy, Jesantha; Truesdale, Todd; Parikh, Rajan; Whitehouse, Kasen; Santra, Sourav; Asher, Damon R; Zaidman, Craig M