Comment
doi: 10.1038/s41584-020-0376-6.
Room for improvement in clinical trials for rare diseases
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- PMID: 31996788
- DOI: 10.1038/s41584-020-0376-6
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Comment
Room for improvement in clinical trials for rare diseases
Nat Rev Rheumatol.
2020 Mar.
No abstract available
Comment on
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Noncompletion and nonpublication of trials studying rare diseases: A cross-sectional analysis.PLoS Med. 2019 Nov 21;16(11):e1002966. doi: 10.1371/journal.pmed.1002966. eCollection 2019 Nov. PLoS Med. 2019. PMID: 31751330 Free PMC article.
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References
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- Rees, C. A. et al. Noncompletion and nonpublication of trials studying rare diseases: a cross-sectional analysis. PLoS Med. 16, e1002966 (2019). - DOI
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- Tatsioni, A. et al. Lost evidence from registered large long-unpublished randomized controlled trials: a survey. Ann. Intern. Med. 171, 300–301 (2019). - DOI
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- Cold Spring Harbor Laboratory medRxiv https://www.medrxiv.org/ (2019).
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- 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
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- 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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