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Figure 4 | Orphanet Journal of Rare Diseases

Figure 4

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

Figure 4

Orphan drug designations for compounds intended to treat lysosomal storage disorders by technology platforms and diseases. N indicates number of orphan drug designations granted. Bold indicates orphan drug designation across two or more technology platforms. “A” indicates disease with one or more FDA approved therapies in the particular technology platform.

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Orphanet Journal of Rare Diseases

ISSN: 1750-1172