Addressing challenges in the diagnosis and treatment of rare genetic diseases
Author:
Publisher
Springer Science and Business Media LLC
Subject
Drug Discovery,Pharmacology,General Medicine
Link
http://www.nature.com/articles/nrd.2017.246.pdf
Reference5 articles.
1. Boycott, K. M. et al. International cooperation to enable the diagnosis of all rare genetic diseases. Am. J. Hum. Genet. 10, 695–705 (2017).
2. Giannuzzi, V. et al. Orphan medicinal products in Europe and United States to cover needs of patients with rare diseases: an increased common effort is to be foreseen. Orphanet J. Rare Dis. 12, 64–75 (2017).
3. Dawkins, H. J. S. et al. Progress in rare diseases research 2010-2016: an IRDiRC perspective. Clin. Transl Sci. http://dx.doi.org/10.1111/cts.12501 (2017).
4. Meekings, K. N., Williams, C. S. & Arrowsmith, J. E. Orphan drug development: an economically viable strategy for biopharma R&D. Drug Disc. Today 17, 660–664 (2012).
5. Austin, C. P. et al. Future of rare diseases research 2017–2027: an IRDiRC perspective. Clin. Transl Sci. http://dx.doi.org/10.1111/cts.12500 (2017).
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