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TITLE: Navigating the orphan medicinal product designation: Evidence requirements for gene therapies in Europe
AUTHORS: Gloria Palomo; Tomas Pose Boirazian; Frauke Naumann Winter; Enrico Costa; Dinah Duarte; Maria Kalland; Eva Malikova; Darius Matusevicius; Dinko Vitezic; Kristina Larsson; Armando Magrelli; Violeta Stoyanova Beninska; Segundo Mariz;
PUBLISHED: 2024, SOURCE: Molecular Therapy
INDEXED IN: Scopus