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Items: 1 to 20 of 64

1.

Perspectives for improving the evaluation and access of therapies for rare lung diseases in Europe.

Luisetti M, Balfour-Lynn IM, Johnson SR, Miravitlles M, Strange C, Trapnell BC, van Bronswijk H, Vogelmeier C.

Respir Med. 2012 Jun;106(6):759-68. doi: 10.1016/j.rmed.2012.02.016. Epub 2012 Mar 11. Review.

2.

[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
3.

Access to information supporting availability of medicines for patients suffering from rare diseases looking for possible treatments: the EuOrphan Service.

Stakisaitis D, Spokiene I, Juskevicius J, Valuckas KP, Baiardi P.

Medicina (Kaunas). 2007;43(6):441-6. Review.

4.

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
5.

Legal assessment of current situation on orphan patients in Lithuania.

Spokiene I.

Medicina (Kaunas). 2008;44(8):571-6. Review.

6.
7.

Rare diseases and orphan drugs: Latvian story.

Logviss K, Krievins D, Purvina S.

Orphanet J Rare Dis. 2014 Sep 18;9:147. doi: 10.1186/s13023-014-0147-z. Review.

8.

Rare diseases social epidemiology: analysis of inequalities.

Kole A, Faurisson F.

Adv Exp Med Biol. 2010;686:223-50. doi: 10.1007/978-90-481-9485-8_14. Review.

PMID:
20824449
9.

[Authorization and reimbursement of orphan drugs in an international comparison].

Roll K, Stargardt T, Schreyögg J.

Gesundheitswesen. 2011 Aug;73(8-9):504-14. doi: 10.1055/s-0030-1262864. Epub 2010 Sep 16. Review. German.

PMID:
20848380
11.

Limitations of drug registries to evaluate orphan medicinal products for the treatment of lysosomal storage disorders.

Hollak CE, Aerts JM, Aymé S, Manuel J.

Orphanet J Rare Dis. 2011 Apr 16;6:16. doi: 10.1186/1750-1172-6-16. Review.

12.

Delayed access to treatments for rare diseases: who's to blame?

Feltmate K, Janiszewski PM, Gingerich S, Cloutier M.

Respirology. 2015 Apr;20(3):361-9. doi: 10.1111/resp.12498. Epub 2015 Feb 26. Review.

13.

Expensive drugs for rare disorders: to treat or not to treat? The case of enzyme replacement therapy for mucopolysaccharidosis VI.

Schlander M, Beck M.

Curr Med Res Opin. 2009 May;25(5):1285-93. doi: 10.1185/03007990902892633 . Review.

PMID:
19366306
14.

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
15.

Drugs for exceptionally rare diseases: do they deserve special status for funding?

Hughes DA, Tunnage B, Yeo ST.

QJM. 2005 Nov;98(11):829-36. Epub 2005 Oct 3. Review.

16.

Review and comparison of clinical evidence submitted to support European Medicines Agency market authorization of orphan-designated oncological treatments.

Winstone J, Chadda S, Ralston S, Sajosi P.

Orphanet J Rare Dis. 2015 Oct 28;10:139. doi: 10.1186/s13023-015-0349-z. Review.

17.

INGN 201: Ad-p53, Ad5CMV-p53, adenoviral p53, p53 gene therapy--introgen, RPR/INGN 201.

[No authors listed]

Drugs R D. 2007;8(3):176-87. Review.

PMID:
17472413
18.

The future in paediatric respirology.

Schmidt HJ, Bhandari V, Bhandari A, Davies J, Marshall BC, Praud JP, Zar HJ, Rubin BK.

Respirology. 2010 Jul;15(5):733-41. doi: 10.1111/j.1440-1843.2010.01745.x. Epub 2010 Apr 8. Review. Erratum in: Respirology. 2010 Oct;15(7):1146.

PMID:
20409021
19.

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
20.

[Rare diseases in pulmonary medicine and its challenges].

Wencker M, Teschler H, Vogelmeier C, Koczulla R.

Pneumologie. 2012 Jul;66(7):437-41. doi: 10.1055/s-0032-1309876. Epub 2012 Jun 12. Review. German.

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