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Fig. 2 | Orphanet Journal of Rare Diseases

Fig. 2

From: Opportunities for developing therapies for rare genetic diseases: focus on gain-of-function and allostery

Fig. 2

Overlap diagrams for the current approved or designated orphan drug space with the rare disease space. Each FDA approved and designated orphan drug was linked to a disease and potential OMIM ID based on the CTD table. 243 and 597 rare diseases are covered by the approved (a) and designed (b) orphan drugs. The current treatment space covers a small fraction of the rare disease space

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