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Items: 1 to 20 of 42

1.

Adopting orphan drugs--two dozen years of treating rare diseases.

Haffner ME.

N Engl J Med. 2006 Feb 2;354(5):445-7. No abstract available.

PMID:
16452556
2.

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
3.
4.

Increasing the use of orphan drugs in clinical practice.

Hulton SA, Greener M.

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

PMID:
15287343
5.
6.

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
7.
8.

Emergence of orphan drugs in the United States: a quantitative assessment of the first 25 years.

Braun MM, Farag-El-Massah S, Xu K, Coté TR.

Nat Rev Drug Discov. 2010 Jul;9(7):519-22. doi: 10.1038/nrd3160. Epub 2010 Jun 7. Review.

PMID:
20531273
9.

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. Review.

PMID:
21060315
10.

Translation of rare disease research into orphan drug development: disease matters.

Heemstra HE, van Weely S, Büller HA, Leufkens HG, de Vrueh RL.

Drug Discov Today. 2009 Dec;14(23-24):1166-73. doi: 10.1016/j.drudis.2009.09.008. Epub 2009 Oct 7. Review.

PMID:
19818412
11.

Rare diseases, drug development, and AIDS: the impact of the Orphan Drug Act.

Arno PS, Bonuck K, Davis M.

Milbank Q. 1995;73(2):231-52. Review.

PMID:
7776947
12.

[Hope for patients with rare diseases--"orphan" drugs].

Kuzelová M, Kubácková K, Palágyi M, Smíd M.

Cas Lek Cesk. 2006;145(4):296-300. Review. Slovak.

PMID:
16639930
13.

1992 Le Tourneau Award. Problems with pharmaceutical regulation in the United States. Drug lag and orphan drugs.

Henry V.

J Leg Med. 1993 Dec;14(4):617-39. Review. No abstract available.

PMID:
8308452
14.

A journey of hope: lessons learned from studies on rare diseases and orphan drugs.

Wästfelt M, Fadeel B, Henter JI.

J Intern Med. 2006 Jul;260(1):1-10. Review.

15.

Infant botulism in the age of botulism immune globulin.

Thompson JA, Filloux FM, Van Orman CB, Swoboda K, Peterson P, Firth SD, Bale JF Jr.

Neurology. 2005 Jun 28;64(12):2029-32. Epub 2005 May 25. Review.

PMID:
15917401
16.

Legal and regulatory aspects of orphan drugs.

Shani S, Yahalom Z.

Pediatr Endocrinol Rev. 2013 Nov;11 Suppl 1:110-5. Review.

PMID:
24380129
17.

Strategic biomarkers for drug development in treating rare diseases and diseases in neonates and infants.

Bai JP, Barrett JS, Burckart GJ, Meibohm B, Sachs HC, Yao L.

AAPS J. 2013 Apr;15(2):447-54. doi: 10.1208/s12248-013-9452-z. Epub 2013 Jan 19. Review.

18.

Drug development for orphan diseases in the context of personalized medicine.

Brewer GJ.

Transl Res. 2009 Dec;154(6):314-22. doi: 10.1016/j.trsl.2009.03.008. Epub 2009 Apr 23. Review.

PMID:
19931198
19.

Drug repositioning for orphan diseases.

Sardana D, Zhu C, Zhang M, Gudivada RC, Yang L, Jegga AG.

Brief Bioinform. 2011 Jul;12(4):346-56. doi: 10.1093/bib/bbr021. Epub 2011 Apr 18. Review.

PMID:
21504985
20.

Proposed 'grant-and-access' program with price caps could stimulate development of drugs for very rare diseases.

Valverde AM, Reed SD, Schulman KA.

Health Aff (Millwood). 2012 Nov;31(11):2528-35. doi: 10.1377/hlthaff.2012.0235. Review.

PMID:
23129684

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