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