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Boosting delivery of rare disease therapies: the IRDiRC Orphan Drug Development Guidebook

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Nature Reviews Drug Discovery 19, 495-496 (2020)



This article was written on behalf of the IRDiRC ODDG Task Force and Therapies Scientific Committee. The members and contributors beyond the authors were A. Aiuti, D. Athanasiou, L. Conklin, S. Cheng, R. Conwit, M. Driessens, M. Haffner, E. Hoffman, S. Jethwa, E. Matsuki, A. Mingorance, T. Morel, A. Pariser, C. Pontes, M. Scarpa and R. Yang. We would also like to thank A.-L. Pham-Hung d’Alexandry d’Orengiani for her support. The work was supported by the European contracts “SUPPORT-IRDiRC” (N° 305207) and “European Joint Programme on Rare Diseases”(N°825575).


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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Competing Interests

The authors declare no competing interests.

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