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Items: 1 to 20 of 120

1.

Advocacy groups and their role in rare diseases research.

Dunkle M, Pines W, Saltonstall PL.

Adv Exp Med Biol. 2010;686:515-25. doi: 10.1007/978-90-481-9485-8_28. Review.

PMID:
20824463
2.

Rare diseases research: expanding collaborative translational research opportunities.

Groft SC.

Chest. 2013 Jul;144(1):16-23. doi: 10.1378/chest.13-0606.

3.

New and evolving rare diseases research programs at the National Institutes of Health.

Groft SC, Rubinstein YR.

Public Health Genomics. 2013;16(6):259-67. doi: 10.1159/000355929. Epub 2014 Feb 3.

4.

Rare diseases, orphan drugs, and orphaned patients.

Scheindlin S.

Mol Interv. 2006 Aug;6(4):186-91. No abstract available.

PMID:
16960139
5.

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

Haffner ME.

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

PMID:
15050976
6.

Novel Treatments for Rare Cancers: The U.S. Orphan Drug Act Is Delivering-A Cross-Sectional Analysis.

Stockklausner C, Lampert A, Hoffmann GF, Ries M.

Oncologist. 2016 Apr;21(4):487-93. doi: 10.1634/theoncologist.2015-0397. Epub 2016 Mar 28.

7.

Navigating through orphan medicinal product regulations in EU and US--similarities and differences.

Tiwari J.

Regul Toxicol Pharmacol. 2015 Feb;71(1):63-7. doi: 10.1016/j.yrtph.2014.11.006. Epub 2014 Dec 8.

PMID:
25497996
8.

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
9.

A cross-national comparative study of orphan drug policies in the United States, the European Union, and Japan: towards a made-in-China orphan drug policy.

Liu BC, He L, He G, He Y.

J Public Health Policy. 2010 Dec;31(4):407-20; discussion 420-1. doi: 10.1057/jphp.2010.30.

PMID:
21119648
10.

Patients mutiny against Orphan Drug Act.

Margolid RE.

Healthspan. 1992 Feb;9(2):25-6. No abstract available.

PMID:
10117860
11.

"Creating hope" and other incentives for drug development for children.

Connor E, Cure P.

Sci Transl Med. 2011 Jan 19;3(66):66cm1. doi: 10.1126/scitranslmed.3001707.

PMID:
21248312
12.

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
13.

Orphan drugs and orphan tests in the USA.

Thoene JG.

Community Genet. 2004;7(4):169-72. No abstract available.

PMID:
15692190
14.

The loneliness of fighting a rare cancer.

Dockser Marcus A.

Health Aff (Millwood). 2010 Jan-Feb;29(1):203-6. doi: 10.1377/hlthaff.2009.0470.

15.

Orphan products development and some methods for maximizing the usefulness of research on rare diseases.

Finkel MJ.

Prog Clin Biol Res. 1985;197:11-8. No abstract available.

PMID:
4070285
16.

How did uncommon disorders become 'rare diseases'? History of a boundary object.

Huyard C.

Sociol Health Illn. 2009 May;31(4):463-77. doi: 10.1111/j.1467-9566.2008.01143.x.

17.

Pressure for drug development in lysosomal storage disorders - a quantitative analysis thirty years beyond the US orphan drug act.

Mechler K, Mountford WK, Hoffmann GF, Ries M.

Orphanet J Rare Dis. 2015 Apr 18;10:46. doi: 10.1186/s13023-015-0262-5.

18.

Rare diseases and orphan drugs.

Taruscio D, Capozzoli F, Frank C.

Ann Ist Super Sanita. 2011;47(1):83-93. doi: 10.4415/ANN_11_01_17. Review.

19.

Expanding research to provide an evidence base for nutritional interventions for the management of inborn errors of metabolism.

Camp KM, Lloyd-Puryear MA, Yao L, Groft SC, Parisi MA, Mulberg A, Gopal-Srivastava R, Cederbaum S, Enns GM, Ershow AG, Frazier DM, Gohagan J, Harding C, Howell RR, Regan K, Stacpoole PW, Venditti C, Vockley J, Watson M, Coates PM.

Mol Genet Metab. 2013 Aug;109(4):319-28. doi: 10.1016/j.ymgme.2013.05.008. Epub 2013 May 23.

20.

Creating a global rare disease patient registry linked to a rare diseases biorepository database: Rare Disease-HUB (RD-HUB).

Rubinstein YR, Groft SC, Bartek R, Brown K, Christensen RA, Collier E, Farber A, Farmer J, Ferguson JH, Forrest CB, Lockhart NC, McCurdy KR, Moore H, Pollen GB, Richesson R, Miller VR, Hull S, Vaught J.

Contemp Clin Trials. 2010 Sep;31(5):394-404. doi: 10.1016/j.cct.2010.06.007. Epub 2010 Jul 8.

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