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Results: 1 to 20 of 94

1.

Abstracts of the 6th European Conference on Rare Diseases and Orphan Products. Brussels, Belgium. May 23-25, 2012.

[No authors listed]

Orphanet J Rare Dis. 2012 Nov 22;7 Suppl 2:A1-41. No abstract available.

PMID:
23210800
[PubMed - indexed for MEDLINE]
Free PMC Article
2.

[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
[PubMed - indexed for MEDLINE]
3.

European regulation on orphan medicinal products: 10 years of experience and future perspectives.

Committee for Orphan Medicinal Products and the European Medicines, Westermark K, Holm BB, Söderholm M, Llinares-Garcia J, Rivière F, Aarum S, Butlen-Ducuing F, Tsigkos S, Wilk-Kachlicka A, N'Diamoi C, Borvendég J, Lyons D, Sepodes B, Bloechl-Daum B, Lhoir A, Todorova M, Kkolos I, Kubáčková K, Bosch-Traberg H, Tillmann V, Saano V, Héron E, Elbers R, Siouti M, Eggenhofer J, Salmon P, Clementi M, Krieviņš D, Matulevičiene A, Metz H, Vincenti AC, Voordouw A, Dembowska-Bagińska B, Nunes AC, Saleh FM, Foltánová T, Možina M, Torrent i Farnell J, Beerman B, Mariz S, Evers MP, Greene L, Thorsteinsson S, Gramstad L, Mavris M, Bignami F, Lorence A, Belorgey C.

Nat Rev Drug Discov. 2011 May;10(5):341-9. doi: 10.1038/nrd3445.

PMID:
21532564
[PubMed - indexed for MEDLINE]
4.

[European incentives for orphan medicinal products].

Enzmann H, Lütz J.

Bundesgesundheitsblatt Gesundheitsforschung Gesundheitsschutz. 2008 May;51(5):500-8. doi: 10.1007/s00103-008-0523-8. Review. German.

PMID:
18696141
[PubMed - indexed for MEDLINE]
5.

[Orphan diseases and orphan medicines: a Belgian and European study].

Denis A, Mergaert L, Fostier C, Cleemput I, Simoens S.

J Pharm Belg. 2009 Dec;(4):131-7. Review. French.

PMID:
20183989
[PubMed - indexed for MEDLINE]
6.

Critical assessment of belgian reimbursement dossiers of orphan drugs.

Denis A, Mergaert L, Fostier C, Cleemput I, Hulstaert F, Simoens S.

Pharmacoeconomics. 2011 Oct;29(10):883-93. doi: 10.2165/11585980-000000000-00000.

PMID:
21905759
[PubMed - indexed for MEDLINE]
7.

Drugs for rare diseases: mixed assessment in Europe.

[No authors listed]

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

PMID:
17323539
[PubMed - indexed for MEDLINE]
8.

Drugs for rare diseases: influence of orphan designation status on price.

Picavet E, Dooms M, Cassiman D, Simoens S.

Appl Health Econ Health Policy. 2011 Jul 1;9(4):275-9. doi: 10.2165/11590170-000000000-00000.

PMID:
21682354
[PubMed - indexed for MEDLINE]
9.

[2nd Eastern-European Conference of Rare Diseases and Their Medications].

[No authors listed]

Orv Hetil. 2007 Apr 1;148(13):619. Hungarian. No abstract available.

PMID:
17383957
[PubMed - indexed for MEDLINE]
10.

The Orphan Drug Act and the development of stem cell-based products for rare diseases.

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

Cell Stem Cell. 2010 Sep 3;7(3):283-7. doi: 10.1016/j.stem.2010.08.003.

PMID:
20804965
[PubMed - indexed for MEDLINE]
Free Article
11.

[Orphan drugs--medications for patients with rare diseases].

Thielke D, Thyssen JP, Hansen BJ.

Ugeskr Laeger. 2006 Jun 5;168(23):2236-8. Danish.

PMID:
16768975
[PubMed - indexed for MEDLINE]
12.

The challenges of orphan drugs and orphan diseases: real and imagined.

Hudson I, Breckenridge A.

Clin Pharmacol Ther. 2012 Aug;92(2):151-3. doi: 10.1038/clpt.2012.72.

PMID:
22814659
[PubMed - indexed for MEDLINE]
13.

[Orphan diseases, rare diseases, time to get mobilised].

Cordier JF.

Rev Prat. 2006 Jan 31;56(2):123-4. Review. French. No abstract available.

PMID:
16584035
[PubMed - indexed for MEDLINE]
14.

[Orphan drugs: availability, reliability and reimbursement].

Kreeftmeijer-Vegter AR, van Veldhuizen CK, de Vries PJ.

Ned Tijdschr Geneeskd. 2012;156(17):A4252. Dutch.

PMID:
22531041
[PubMed - indexed for MEDLINE]
15.

Predictors of orphan drug approval in the European Union.

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

Eur J Clin Pharmacol. 2008 May;64(5):545-52. doi: 10.1007/s00228-007-0454-6. Epub 2008 Jan 22.

PMID:
18210097
[PubMed - indexed for MEDLINE]
Free PMC Article
16.

Orphan products: hope for people with rare diseases.

Rados C.

FDA Consum. 2003 Nov-Dec;37(6):10-5. No abstract available.

PMID:
14986582
[PubMed - indexed for MEDLINE]
Free Article
17.

Regulatory considerations for developing drugs for rare diseases: orphan designations and early phase clinical trials.

Pariser AR, Xu K, Milto J, Coté TR.

Discov Med. 2011 Apr;11(59):367-75. Review.

PMID:
21524390
[PubMed - indexed for MEDLINE]
Free Article
18.

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
[PubMed - indexed for MEDLINE]
19.

The Food and Drug Administration's Office of Orphan Products Development: incentives, grants, and special designations speed therapies for orphan diseases.

Haffner ME.

Retina. 2005 Dec;25(8 Suppl):S89-S90. Review. No abstract available.

PMID:
16374359
[PubMed - indexed for MEDLINE]
20.

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
[PubMed - indexed for MEDLINE]

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