Effects of gene replacement therapy with resamirigene bilparvovec (AT132) on skeletal muscle pathology in X-linked myotubular myopathy: results from a substudy of the ASPIRO open-label clinical trial
使用 resamirigene bilparvovec (AT132) 进行基因替代疗法对 X 连锁肌管性肌病骨骼肌病理的影响:来自 ASPIRO 开放标签临床试验子研究的结果
期刊:EBioMedicine
影响因子:9.7
doi:10.1016/j.ebiom.2023.104894
Michael W Lawlor, Benedikt Schoser, Marta Margeta, Caroline A Sewry, Karra A Jones, Perry B Shieh, Nancy L Kuntz, Barbara K Smith, James J Dowling, Wolfgang Müller-Felber, Carsten G Bönnemann, Andreea M Seferian, Astrid Blaschek, Sarah Neuhaus, A Reghan Foley, Dimah N Saade, Etsuko Tsuchiya, Ummulwa