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Items: 10

1.

The availability and affordability of orphan drugs for rare diseases in China.

Gong S, Wang Y, Pan X, Zhang L, Huang R, Chen X, Hu J, Xu Y, Jin S.

Orphanet J Rare Dis. 2016 Feb 27;11:20. doi: 10.1186/s13023-016-0392-4.

2.

Increased Probability of Co-Occurrence of Two Rare Diseases in Consanguineous Families and Resolution of a Complex Phenotype by Next Generation Sequencing.

Lal D, Neubauer BA, Toliat MR, Altmüller J, Thiele H, Nürnberg P, Kamrath C, Schänzer A, Sander T, Hahn A, Nothnagel M.

PLoS One. 2016 Jan 20;11(1):e0146040. doi: 10.1371/journal.pone.0146040. eCollection 2016.

3.

Contribution of Electronic Medical Records to the Management of Rare Diseases.

Bremond-Gignac D, Lewandowski E, Copin H.

Biomed Res Int. 2015;2015:954283. doi: 10.1155/2015/954283. Epub 2015 Oct 11.

4.

Rare diseases are a 'common' problem for clinicians.

Elliott E, Zurynski Y.

Aust Fam Physician. 2015 Sep;44(9):630-3.

5.

Innovative research methods for studying treatments for rare diseases: methodological review.

Gagne JJ, Thompson L, O'Keefe K, Kesselheim AS.

BMJ. 2014 Nov 24;349:g6802. doi: 10.1136/bmj.g6802. Review.

6.

Social media methods for studying rare diseases.

Schumacher KR, Stringer KA, Donohue JE, Yu S, Shaver A, Caruthers RL, Zikmund-Fisher BJ, Fifer C, Goldberg C, Russell MW.

Pediatrics. 2014 May;133(5):e1345-53. doi: 10.1542/peds.2013-2966. Epub 2014 Apr 14.

7.

Strategies for postmarketing surveillance of drugs for rare diseases.

Kesselheim AS, Gagne JJ.

Clin Pharmacol Ther. 2014 Mar;95(3):265-8. doi: 10.1038/clpt.2013.218. Epub 2013 Nov 5. Review.

PMID:
24193169
8.

Cross-sector sponsorship of research in eosinophilic esophagitis: a collaborative model for rational drug development in rare diseases.

Fiorentino R, Liu G, Pariser AR, Mulberg AE.

J Allergy Clin Immunol. 2012 Sep;130(3):613-6. doi: 10.1016/j.jaci.2012.07.011. Epub 2012 Jul 31.

PMID:
22857796
9.

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.

PMID:
22371464
10.

'Doctor Google' ending the diagnostic odyssey in lysosomal storage disorders: parents using internet search engines as an efficient diagnostic strategy in rare diseases.

Bouwman MG, Teunissen QG, Wijburg FA, Linthorst GE.

Arch Dis Child. 2010 Aug;95(8):642-4. doi: 10.1136/adc.2009.171827. Epub 2010 Apr 23.

PMID:
20418338

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