Author:
Yin Hao,Kauffman Kevin J.,Anderson Daniel G.
Publisher
Springer Science and Business Media LLC
Subject
Drug Discovery,Pharmacology,General Medicine
Reference146 articles.
1. Boycott, K. M., Vanstone, M. R., Bulman, D. E. & MacKenzie, A. E. Rare-disease genetics in the era of next-generation sequencing: discovery to translation. Nat. Rev. Genet. 14, 681–691 (2013).
2. Pharmaceutical Research and Manufacturers of America. Medicines In Development. Rare Diseases: A Report On Orphan Drugs In The Pipeline Presented By America's Biopharmaceutical Research Companies. PhRMA.org
http://phrma-docs.phrma.org/sites/default/files/pdf/Rare_Diseases_2013.pdf
(2013).
3. Elborn, J. S. Cystic fibrosis. Lancet 388, 2519–2531 (2016).
4. Yang, Q. Small molecule therapy for genetic diseases. Yale J. Biol. Med. 85, 161–162 (2012).
5. O'Connor, T. P. & Crystal, R. G. Genetic medicines: treatment strategies for hereditary disorders. Nat. Rev. Genet. 7, 261–276 (2006).
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