Novel orphan medicines and abandoned pathways - the US Orphan Drug Act of 1983 and the impact on rare rheumatologic diseases and lysosomal storage disorders

Ries, Markus; Lutz, Thomas; Lampert, Anette; Mountford, William; Mechler, Konstantin; Hoffmann, Georg F

doi:10.1186/2194-7791-2-S1-A1

Molecular and Cellular Pediatrics

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Novel orphan medicines and abandoned pathways - the US Orphan Drug Act of 1983 and the impact on rare rheumatologic diseases and lysosomal storage disorders

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