565 related articles for article (PubMed ID: 22371464)
1. 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. PubMed ID: 22371464
[TBL] [Abstract][Full Text] [Related]
2. Therapies for inborn errors of metabolism: what has the orphan drug act delivered?
Talele SS; Xu K; Pariser AR; Braun MM; Farag-El-Massah S; Phillips MI; Thompson BH; Coté TR
Pediatrics; 2010 Jul; 126(1):101-6. PubMed ID: 20566615
[TBL] [Abstract][Full Text] [Related]
3. The US Orphan Drug Act: rare disease research stimulator or commercial opportunity?
Wellman-Labadie O; Zhou Y
Health Policy; 2010 May; 95(2-3):216-28. PubMed ID: 20036435
[TBL] [Abstract][Full Text] [Related]
4. Database identifies FDA-approved drugs with potential to be repurposed for treatment of orphan diseases.
Xu K; Coté TR
Brief Bioinform; 2011 Jul; 12(4):341-5. PubMed ID: 21357612
[TBL] [Abstract][Full Text] [Related]
5. Developing treatments for inborn errors: incentives available to the clinician.
Haffner ME
Mol Genet Metab; 2004 Apr; 81 Suppl 1():S63-6. PubMed ID: 15050976
[TBL] [Abstract][Full Text] [Related]
6. Ethical imperatives of timely access to orphan drugs: is possible to reconcile economic incentives and patients' health needs?
Rodriguez-Monguio R; Spargo T; Seoane-Vazquez E
Orphanet J Rare Dis; 2017 Jan; 12(1):1. PubMed ID: 28057032
[TBL] [Abstract][Full Text] [Related]
7. 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. PubMed ID: 20856241
[TBL] [Abstract][Full Text] [Related]
8. 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. PubMed ID: 21524390
[TBL] [Abstract][Full Text] [Related]
9. Emergence of orphan drugs in the United States: a quantitative assessment of the first 25 years.
Braun MM; Farag-El-Massah S; Xu K; Coté TR
Nat Rev Drug Discov; 2010 Jul; 9(7):519-22. PubMed ID: 20531273
[TBL] [Abstract][Full Text] [Related]
10. Orphan medicinal products in Europe and United States to cover needs of patients with rare diseases: an increased common effort is to be foreseen.
Giannuzzi V; Conte R; Landi A; Ottomano SA; Bonifazi D; Baiardi P; Bonifazi F; Ceci A
Orphanet J Rare Dis; 2017 Apr; 12(1):64. PubMed ID: 28372595
[TBL] [Abstract][Full Text] [Related]
11. Using four decades of FDA orphan drug designations to describe trends in rare disease drug development: substantial growth seen in development of drugs for rare oncologic, neurologic, and pediatric-onset diseases.
Miller KL; Fermaglich LJ; Maynard J
Orphanet J Rare Dis; 2021 Jun; 16(1):265. PubMed ID: 34107994
[TBL] [Abstract][Full Text] [Related]
12. The impact of the Orphan Drug Act on Food and Drug Administration-approved therapies for rare skin diseases and skin-related cancers.
Karas L; Lu CY; Agrawal PB; Asgari MM
J Am Acad Dermatol; 2019 Sep; 81(3):867-877. PubMed ID: 31103566
[TBL] [Abstract][Full Text] [Related]
13. "Creating hope" and other incentives for drug development for children.
Connor E; Cure P
Sci Transl Med; 2011 Jan; 3(66):66cm1. PubMed ID: 21248312
[TBL] [Abstract][Full Text] [Related]
14. [Hope for patients with rare diseases--"orphan" drugs].
Kuzelová M; Kubácková K; Palágyi M; Smíd M
Cas Lek Cesk; 2006; 145(4):296-300. PubMed ID: 16639930
[TBL] [Abstract][Full Text] [Related]
15. Characteristics of orphan drug applications that fail to achieve marketing approval in the USA.
Heemstra HE; Leufkens HG; Rodgers RP; Xu K; Voordouw BC; Braun MM
Drug Discov Today; 2011 Jan; 16(1-2):73-80. PubMed ID: 21094692
[TBL] [Abstract][Full Text] [Related]
16. Incentivizing Orphan Product Development: United States Food and Drug Administration Orphan Incentive Programs.
Le TT
Adv Exp Med Biol; 2017; 1031():183-196. PubMed ID: 29214572
[TBL] [Abstract][Full Text] [Related]
17. 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. PubMed ID: 27022038
[TBL] [Abstract][Full Text] [Related]
18. Investigating the landscape of US orphan product approvals.
Miller KL; Lanthier M
Orphanet J Rare Dis; 2018 Oct; 13(1):183. PubMed ID: 30348193
[TBL] [Abstract][Full Text] [Related]
19. Drugs for rare diseases: mixed assessment in Europe.
Prescrire Int; 2007 Feb; 16(87):36-42. PubMed ID: 17323539
[TBL] [Abstract][Full Text] [Related]
20. Precision Medicines' Impact on Orphan Drug Designation.
Mueller CM; Rao GR; Miller Needleman KI
Clin Transl Sci; 2019 Nov; 12(6):633-640. PubMed ID: 31297924
[TBL] [Abstract][Full Text] [Related]
[Next] [New Search]