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. 2017 Feb 8;35(2):117-118.
doi: 10.1038/nbt.3787.

Industrializing rare disease therapy discovery and development

Sean Ekins  1
Affiliations

Industrializing rare disease therapy discovery and development

Sean Ekins. Nat Biotechnol. .
No abstract available

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Figures

Figure 1
Figure 1
Schematic of a ‘production line’ for developing gene therapies, protein replacement and small molecule approaches for rare diseases in which researchers, clinicians, patients and advocates collaborate to bring different treatments for different rare diseases to the point of clinical trials.
See this image and copyright information in PMC

References

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