This site needs JavaScript to work properly. Please enable it to take advantage of the complete set of features!

Skip to main page content

Add to Collections

Your saved search

Name of saved search:

Search terms:

Test search terms

Would you like email updates of new search results?

Yes
No

Email: (change)

Frequency:

Which day?

Which day?

Report format:

Send at most:

Send even when there aren't any new results

Optional text in email:

Your RSS Feed

Comment

. 2006 Feb 2;354(5):445-7.

doi: 10.1056/NEJMp058317.

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

Marlene E Haffner¹

Affiliations

PMID: 16452556
DOI: 10.1056/NEJMp058317

Comment

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

Marlene E Haffner. N Engl J Med. 2006.

. 2006 Feb 2;354(5):445-7.

doi: 10.1056/NEJMp058317.

Author

Marlene E Haffner¹

Affiliation

¹ Office of Orphan Products Development at the Food and Drug Administration, Rockville, Md, USA.

PMID: 16452556
DOI: 10.1056/NEJMp058317

No abstract available

PubMed Disclaimer

Comment on

Human botulism immune globulin for the treatment of infant botulism.
Arnon SS, Schechter R, Maslanka SE, Jewell NP, Hatheway CL. Arnon SS, et al. N Engl J Med. 2006 Feb 2;354(5):462-71. doi: 10.1056/NEJMoa051926. N Engl J Med. 2006. PMID: 16452558 Clinical Trial.

Similar articles

Rare diseases, orphan drugs, and orphaned patients.
Scheindlin S. Scheindlin S. Mol Interv. 2006 Aug;6(4):186-91. doi: 10.1124/mi.6.4.2. Mol Interv. 2006. PMID: 16960139 No abstract available.
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. Burke KA, et al. Clin Pharmacol Ther. 2010 Oct;88(4):449-53. doi: 10.1038/clpt.2010.193. Clin Pharmacol Ther. 2010. PMID: 20856241 Review.
Orphan drugs and orphan tests in the USA.
Thoene JG. Thoene JG. Community Genet. 2004;7(4):169-72. doi: 10.1159/000082255. Community Genet. 2004. PMID: 15692190 No abstract available.
Rare opportunities appear on the horizon to treat rare diseases.
Torres C. Torres C. Nat Med. 2010 Mar;16(3):241. doi: 10.1038/nm0310-241. Nat Med. 2010. PMID: 20208483 No abstract available.
Creation and development of the public service orphan drug Human Botulism Immune Globulin.
Arnon SS. Arnon SS. Pediatrics. 2007 Apr;119(4):785-9. doi: 10.1542/peds.2006-0646. Pediatrics. 2007. PMID: 17403850 Review.

See all similar articles

Cited by

Incentives for orphan drug research and development in the United States.
Seoane-Vazquez E, Rodriguez-Monguio R, Szeinbach SL, Visaria J. Seoane-Vazquez E, et al. Orphanet J Rare Dis. 2008 Dec 16;3:33. doi: 10.1186/1750-1172-3-33. Orphanet J Rare Dis. 2008. PMID: 19087348 Free PMC article.
Comparison of EQ-5D and SF-6D utilities in Pompe disease.
Kanters TA, Redekop WK, Kruijshaar ME, van der Ploeg AT, Rutten-van Mölken MP, Hakkaart L. Kanters TA, et al. Qual Life Res. 2015 Apr;24(4):837-44. doi: 10.1007/s11136-014-0833-2. Epub 2014 Oct 24. Qual Life Res. 2015. PMID: 25342117
Emergence of pediatric rare diseases: Review of present policies and opportunities for improvement.
Bavisetty S, Grody WW, Yazdani S. Bavisetty S, et al. Rare Dis. 2013 Jan 28;1:e23579. doi: 10.4161/rdis.23579. eCollection 2013. Rare Dis. 2013. PMID: 25002987 Free PMC article. Review.
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. Stockklausner C, et al. Oncologist. 2016 Apr;21(4):487-93. doi: 10.1634/theoncologist.2015-0397. Epub 2016 Mar 28. Oncologist. 2016. PMID: 27022038 Free PMC article.
Drug discovery and development for rare genetic disorders.
Sun W, Zheng W, Simeonov A. Sun W, et al. Am J Med Genet A. 2017 Sep;173(9):2307-2322. doi: 10.1002/ajmg.a.38326. Epub 2017 Jul 21. Am J Med Genet A. 2017. PMID: 28731526 Free PMC article. Review.

See all "Cited by" articles

Publication types

Actions
Actions

MeSH terms

Actions
Actions
Actions
Actions
Actions
Actions
Actions
Actions
Actions
Actions
Actions
Actions
Actions
Actions

Substances

Actions
Actions
Actions

LinkOut - more resources

Full Text Sources
- Atypon
- Ovid Technologies, Inc.
Medical
- MedlinePlus Health Information