Navigating the orphan medicinal product designation: Evidence requirements for gene therapies in Europe
孤儿药认定指南:欧洲基因疗法的证据要求
期刊:Molecular Therapy
影响因子:12
doi:10.1016/j.ymthe.2024.10.015
Palomo, Gloria M; Pose-Boirazian, Tomas; Naumann-Winter, Frauke; Costa, Enrico; Duarte, Dinah M; Kalland, Maria E; Malikova, Eva; Matusevicius, Darius; Vitezic, Dinko; Larsson, Kristina; Magrelli, Armando; Stoyanova-Beninska, Violeta; Mariz, Segundo