Room for improvement in clinical trials for rare diseases
Author:
Publisher
Springer Science and Business Media LLC
Subject
Rheumatology
Link
http://www.nature.com/articles/s41584-020-0376-6.pdf
Reference10 articles.
1. Rees, C. A. et al. Noncompletion and nonpublication of trials studying rare diseases: a cross-sectional analysis. PLoS Med. 16, e1002966 (2019).
2. Tatsioni, A. et al. Lost evidence from registered large long-unpublished randomized controlled trials: a survey. Ann. Intern. Med. 171, 300–301 (2019).
3. Cold Spring Harbor Laboratory medRxiv https://www.medrxiv.org/ (2019).
4. 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).
5. 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).
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