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
期刊:Nature Medicine
影响因子:50
doi:10.1038/s41591-024-03304-z
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