Abstract
Interest in developing drugs for rare diseases has increased substantially in recent years. This article from the US Food and Drug Administration highlights the role of regulators in catalysing further progress in this field.
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The use or generation of biomedical data and existing medicines to discover and establish new treatments for patients with rare diseases – recommendations of the IRDiRC Data Mining and Repurposing Task Force
Orphanet Journal of Rare Diseases Open Access 15 October 2019
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References
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Coté, T., Xu, K. & Pariser, A. Accelerating orphan drug development. Nat Rev Drug Discov 9, 901–902 (2010). https://doi.org/10.1038/nrd3340
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DOI: https://doi.org/10.1038/nrd3340
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