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Comment
. 2020 Mar;16(3):131-132.
doi: 10.1038/s41584-020-0376-6.

Room for improvement in clinical trials for rare diseases

Anne Musters  1   2 , Sander W Tas  3   4
Affiliations
Comment

Room for improvement in clinical trials for rare diseases

Anne Musters et al. Nat Rev Rheumatol. 2020 Mar.
No abstract available

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References

    1. Rees, C. A. et al. Noncompletion and nonpublication of trials studying rare diseases: a cross-sectional analysis. PLoS Med. 16, e1002966 (2019). - DOI
    1. Tatsioni, A. et al. Lost evidence from registered large long-unpublished randomized controlled trials: a survey. Ann. Intern. Med. 171, 300–301 (2019). - DOI
    1. Cold Spring Harbor Laboratory medRxiv https://www.medrxiv.org/ (2019).
    1. Musters, A. et al. Discovery of innovative therapies for rare immune-mediated inflammatory diseases via off-label prescription of biologics: the case of IL-6 receptor blockade in Castleman’s disease. Front. Immunol. 6, 625 (2015). - DOI
    1. Fajgenbaum, D. C. et al. Identifying and targeting pathogenic PI3K/AKT/mTOR signaling in IL-6-blockade-refractory idiopathic multicentric Castleman disease. J. Clin. Invest. 130, 4451–4463 (2019). - DOI

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