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Time for a new approach to drug development for rare systemic autoinflammatory diseases

Nature Reviews Rheumatology (2024)Cite this article

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Despite the transformative potential of treatments for systemic autoinflammatory diseases, these conditions remain underfunded and understudied. Urgent, coordinated action among stakeholders is needed to overcome regulatory and research barriers, as is innovation and advocacy for the development of effective therapies for these rare diseases.

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Acknowledgements

E.D. and R.G.M. are co-founders of TARN, N.M.Z. is a coordinator of TARN, and M.R. is a member of its steering committee. The authors would like to acknowledge other core steering committee members of TARN: S. Canna, F. Dedeoglu and K. Durrant. We also extend our gratitude to F. Garcia-Bournissen for invaluable contributions to the conceptualization of this Comment, which were pivotal in refining the arguments and perspectives explored.

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Authors and Affiliations

  1. Department of Pediatrics, Schulich School of Medicine & Dentistry, Western University, London, Ontario, Canada

    Natalie M. Zitoun, Erkan Demirkaya & Micol Romano

  2. Canadian Behcet’s and Autoinflammatory Diseases Center, University of Western Ontario, London, Ontario, Canada

    Erkan Demirkaya & Micol Romano

  3. Translational Autoinflammatory Diseases Section, Laboratory of Clinical Immunology and Microbiology, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, MD, USA

    Raphaela Goldbach-Mansky

Authors
  1. Natalie M. Zitoun
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  2. Erkan Demirkaya
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  3. Raphaela Goldbach-Mansky
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  4. Micol Romano
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Correspondence to Micol Romano.

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The authors declare no competing interests.

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Zitoun, N.M., Demirkaya, E., Goldbach-Mansky, R. et al. Time for a new approach to drug development for rare systemic autoinflammatory diseases. Nat Rev Rheumatol (2024). https://doi.org/10.1038/s41584-024-01108-6

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