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10.4155/fmc.14.65 http://scihub22266oqcxt.onion/10.4155/fmc.14.65 C4354801!4354801!25068983     free     free     free       Future+Med+Chem 2014 ; 6 (9): 987-1002 Nephropedia Template TP {{tp|p=25068983|t=2014. The discovery of medicines for rare diseases |pdf=|usr=}}{{25068983}} gab.com Text The discovery of medicines for rare diseases JO: Future Med Chem http://www.kidney.de/pget.php?&tw=1&pmid=25068983 #FOAvip There is a pressing need for new medicines (new molecular entities; NMEs) for rare diseases as few of the 6800 rare diseases (according to the NIH) have approved treatments. Drug discovery strategies for the 102 orphan NMEs approved by the US FDA between 1999 and 2012 were analyzed to learn from past success: 46 NMEs were first in class; 51 were followers; and five were imaging agents. First-in-class medicines were discovered with phenotypic assays (15), target-based approaches (12) and biologic strategies (18). Identification of genetic causes in areas with more basic and translational research such as cancer and in-born errors in metabolism contributed to success regardless of discovery strategy. In conclusion, greater knowledge increases the chance of success and empirical solutions can be effective when knowledge is incomplete. Twit Text FOAVip The discovery of medicines for rare diseases ????Future Med Chem http://www.kidney.de/pget.php?&tw=1&pmid=25068983 #FOAvip Twit Text # Free Paper on # # # # # LINK http://www.kidney.de/pget.php?&tw=1&pmid=25068983 #FOAvip English Wikipedia <ref>{{Cite pmid|25068983}}</ref> The discovery of medicines for rare diseases #MMPMID25068983 Swinney DC; Xia S Future Med Chem 2014[Jun]; 6 (9): 987-1002 PMID25068983show ga There is a pressing need for new medicines (new molecular entities; NMEs) for rare diseases as few of the 6800 rare diseases (according to the NIH) have approved treatments. Drug discovery strategies for the 102 orphan NMEs approved by the US FDA between 1999 and 2012 were analyzed to learn from past success: 46 NMEs were first in class; 51 were followers; and five were imaging agents. First-in-class medicines were discovered with phenotypic assays (15), target-based approaches (12) and biologic strategies (18). Identification of genetic causes in areas with more basic and translational research such as cancer and in-born errors in metabolism contributed to success regardless of discovery strategy. In conclusion, greater knowledge increases the chance of success and empirical solutions can be effective when knowledge is incomplete. äThe discovery of medicines for rare diseases (epmc).pdf DeepDyve Pubget Overpricing