INCEPTUS Natural History, Run-in Study for Gene Replacement Clinical Trial in X-Linked Myotubular Myopathy
INCEPTUS 自然史,X连锁肌管性肌病基因替代临床试验的导入研究
期刊:Journal of Neuromuscular Diseases
影响因子:3.4
doi:10.3233/JND-210781
Dowling, James J; Müller-Felber, Wolfgang; Smith, Barbara K; Bönnemann, Carsten G; Kuntz, Nancy L; Muntoni, Francesco; Servais, Laurent; Alfano, Lindsay N; Beggs, Alan H; Bilder, Deborah A; Blaschek, Astrid; Duong, Tina; Graham, Robert J; Jain, Minal; Lawlor, Michael W; Lee, Jun; Coats, Julie; Lilien, Charlotte; Lowes, Linda P; MacBean, Victoria; Neuhaus, Sarah; Noursalehi, Mojtaba; Pitts, Teresa; Finlay, Caroline; Christensen, Sarah; Rafferty, Gerrard; Seferian, Andreea M; Tsuchiya, Etsuko; James, Emma S; Miller, Weston; Sepulveda, Bryan; Vila, Maria Candida; Prasad, Suyash; Rico, Salvador; Shieh, Perry B