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Items: 1 to 20 of 342

1.
2.

Developing treatments for inborn errors: incentives available to the clinician.

Haffner ME.

Mol Genet Metab. 2004 Apr;81 Suppl 1:S63-6.

PMID:
15050976
3.

The US Orphan Drug Act: rare disease research stimulator or commercial opportunity?

Wellman-Labadie O, Zhou Y.

Health Policy. 2010 May;95(2-3):216-28. doi: 10.1016/j.healthpol.2009.12.001. Epub 2009 Dec 29.

PMID:
20036435
5.

A regulatory overview about rare diseases.

Llinares J.

Adv Exp Med Biol. 2010;686:193-207. doi: 10.1007/978-90-481-9485-8_12. Review.

PMID:
20824447
6.

What the Orphan Drug Act has done lately for children with rare diseases: a 10-year analysis.

Thorat C, Xu K, Freeman SN, Bonnel RA, Joseph F, Phillips MI, Imoisili MA.

Pediatrics. 2012 Mar;129(3):516-21. doi: 10.1542/peds.2011-1798. Epub 2012 Feb 27.

7.

The economics of orphan drug policy in the US. Can the legislation be improved?

Peabody JW, Ruby A, Cannon P.

Pharmacoeconomics. 1995 Nov;8(5):374-84. Review.

PMID:
10160072
8.

Accelerating access to treatments for rare diseases.

Dunoyer M.

Nat Rev Drug Discov. 2011 Jun 24;10(7):475-6. doi: 10.1038/nrd3493.

PMID:
21701499
9.

Orphan drugs, orphan diseases. The first decade of orphan drug legislation in the EU.

Joppi R, Bertele' V, Garattini S.

Eur J Clin Pharmacol. 2013 Apr;69(4):1009-24. doi: 10.1007/s00228-012-1423-2. Epub 2012 Oct 23.

PMID:
23090701
10.

Raising orphans: how clinical development programs of drugs for rare and common diseases are different.

Orfali M, Feldman L, Bhattacharjee V, Harkins P, Kadam S, Lo C, Ravi M, Shringarpure DT, Mardekian J, Cassino C, Coté T.

Clin Pharmacol Ther. 2012 Aug;92(2):262-4. doi: 10.1038/clpt.2012.87. Epub 2012 Jun 27.

PMID:
22739137
11.

Drugs for rare diseases: mixed assessment in Europe.

[No authors listed]

Prescrire Int. 2007 Feb;16(87):36-42.

PMID:
17323539
12.

Clinical pharmacology as a cornerstone of orphan drug development.

Bashaw ED, Huang SM, Coté TR, Pariser AR, Garnett CE, Burckart G, Zhang L, Men AY, Le CD, Charlab R, Gobburu JV, Lesko LJ.

Nat Rev Drug Discov. 2011 Oct 31;10(11):795-6. doi: 10.1038/nrd3595. Review.

PMID:
22037026
13.

The US orphan drug programme 1983-1995.

Shulman SR, Manocchia M.

Pharmacoeconomics. 1997 Sep;12(3):312-26. Review.

PMID:
10170457
14.

Incentives for orphan drug research and development in the United States.

Seoane-Vazquez E, Rodriguez-Monguio R, Szeinbach SL, Visaria J.

Orphanet J Rare Dis. 2008 Dec 16;3:33. doi: 10.1186/1750-1172-3-33.

15.
16.

The impact of the Orphan Drug Act on the development and advancement of neurological products for rare diseases: a descriptive review.

Burke KA, Freeman SN, Imoisili MA, Coté TR.

Clin Pharmacol Ther. 2010 Oct;88(4):449-53. doi: 10.1038/clpt.2010.193. Review.

PMID:
20856241
17.

Characteristics of orphan drug applications that fail to achieve marketing approval in the USA.

Heemstra HE, Leufkens HG, Rodgers RP, Xu K, Voordouw BC, Braun MM.

Drug Discov Today. 2011 Jan;16(1-2):73-80. doi: 10.1016/j.drudis.2010.11.006. Epub 2010 Nov 20. Review.

PMID:
21094692
18.

Increasing the use of orphan drugs in clinical practice.

Hulton SA, Greener M.

Hosp Med. 2004 Jul;65(7):400-3. Review.

PMID:
15287343
19.

Economic considerations in the provision of treatments for rare diseases.

McCabe C, Edlin R, Round J.

Adv Exp Med Biol. 2010;686:211-22. doi: 10.1007/978-90-481-9485-8_13. Review.

PMID:
20824448
20.

Rare diseases, orphan drugs and their regulation: questions and misconceptions.

Tambuyzer E.

Nat Rev Drug Discov. 2010 Dec;9(12):921-9. doi: 10.1038/nrd3275. Epub 2010 Nov 9.

PMID:
21060315
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