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IRDiRC Drug Repurposing Guidebook: making better use of existing drugs to tackle rare diseases

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Nature Reviews Drug Discovery 22, 937-938 (2023)



This article was written on behalf of the IRDiRC DRG Task Force and Therapies Scientific Committee. The members and contributors beyond the authors were Maria Cavaller Belaubi, Luca Falciola, Christine Fetro, Maria Gogou, Florence Guillot, Virginie Hivert, Peter-Bram ’t Hoen, Sonsoles Hortelano Blanco, Oxana Iliach, Ania Korsunska, Ramaiah Muthyala, Vinciane Pirard, Ken Sakushima, Marco Schmidt and Nivedita Valentine. We would also like to thank Galliano Zanello for his support. The work was supported by the European contract “European Joint Programme on Rare Diseases”(N°825575).


  1. Dawkins, H. J. S. et al. Progress in Rare Diseases Research 2010–2016: An IRDiRC Perspective. Clin. Transl. Sci. 11, 11–20 (2018).

    Article  PubMed  Google Scholar 

  2. Fang, H. et al. FDALabel for drug repurposing studies and beyond. Nat. Biotechnol. 38, 1378–1379 (2020).

    Article  PubMed  Google Scholar 

  3. Austin, C. P., Mount, B. A. & Colvis, C. M. Envisioning an actionable research agenda to facilitate repurposing of off-patent drugs. Nat. Rev. Drug Discov. 20, 723–724 (2021).

    Article  PubMed  Google Scholar 

  4. Hechtelt Jonker, A. et al. Boosting delivery of rare disease therapies: the IRDiRC Orphan Drug Development Guidebook. Nat. Rev. Drug Discov. 19, 495–496 (2020).

    Article  PubMed  Google Scholar 

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Supplementary Information

  1. 41573_2023_CM26185128_MOESM1_ESM

Competing Interests

The authors declare no competing interests. The views expressed in this article are the personal views of the author(s) and may not be understood as being made on behalf of their employer(s)/organization(s).

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