Pressure for drug development in lysosomal storage disorders – a quantitative analysis thirty years beyond the US orphan drug act

Pressure for drug development in lysosomal storage disorders – a quantitative analysis thirty years beyond the US orphan drug act. Konstantin Mechler, William K Mountford, Georg F Hoffmann and Markus Ries; Orphanet Journal of Rare Diseases 2015, 10:46 doi:10.1186/s13023-015-0262-5

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