These tools will no longer be maintained as of December 31, 2024. Archived website can be found here. PubMed4Hh GitHub repository can be found here. Contact NLM Customer Service if you have questions.


BIOMARKERS

Molecular Biopsy of Human Tumors

- a resource for Precision Medicine *

252 related articles for article (PubMed ID: 22704758)

  • 1. A generalizable pre-clinical research approach for orphan disease therapy.
    Beaulieu CL; Samuels ME; Ekins S; McMaster CR; Edwards AM; Krainer AR; Hicks GG; Frey BJ; Boycott KM; Mackenzie AE
    Orphanet J Rare Dis; 2012 Jun; 7():39. PubMed ID: 22704758
    [TBL] [Abstract][Full Text] [Related]  

  • 2. New approaches to the treatment of orphan genetic disorders: Mitigating molecular pathologies using chemicals.
    Velho RV; Sperb-Ludwig F; Schwartz IV
    An Acad Bras Cienc; 2015 Aug; 87(2 Suppl):1375-88. PubMed ID: 26247150
    [TBL] [Abstract][Full Text] [Related]  

  • 3. Opportunities for developing therapies for rare genetic diseases: focus on gain-of-function and allostery.
    Chen B; Altman RB
    Orphanet J Rare Dis; 2017 Apr; 12(1):61. PubMed ID: 28412959
    [TBL] [Abstract][Full Text] [Related]  

  • 4. Orphan diseases: state of the drug discovery art.
    Volmar CH; Wahlestedt C; Brothers SP
    Wien Med Wochenschr; 2017 Jun; 167(9-10):197-204. PubMed ID: 26819216
    [TBL] [Abstract][Full Text] [Related]  

  • 5. Drug discovery and development for rare genetic disorders.
    Sun W; Zheng W; Simeonov A
    Am J Med Genet A; 2017 Sep; 173(9):2307-2322. PubMed ID: 28731526
    [TBL] [Abstract][Full Text] [Related]  

  • 6. Challenges in orphan drug development and regulatory policy in China.
    Cheng A; Xie Z
    Orphanet J Rare Dis; 2017 Jan; 12(1):13. PubMed ID: 28100254
    [TBL] [Abstract][Full Text] [Related]  

  • 7. eRepo-ORP: Exploring the Opportunity Space to Combat Orphan Diseases with Existing Drugs.
    Brylinski M; Naderi M; Govindaraj RG; Lemoine J
    J Mol Biol; 2018 Jul; 430(15):2266-2273. PubMed ID: 29237557
    [TBL] [Abstract][Full Text] [Related]  

  • 8. Orphan Drugs and Their Impact on Pharmaceutical Development.
    Attwood MM; Rask-Andersen M; Schiöth HB
    Trends Pharmacol Sci; 2018 Jun; 39(6):525-535. PubMed ID: 29779531
    [TBL] [Abstract][Full Text] [Related]  

  • 9. Rare Diseases: Drug Discovery and Informatics Resource.
    Zhao M; Wei DQ
    Interdiscip Sci; 2018 Mar; 10(1):195-204. PubMed ID: 29094320
    [TBL] [Abstract][Full Text] [Related]  

  • 10. Managing drugs for rare genetic diseases: trends and insights.
    Zitter M
    Manag Care; 2005 Feb; 14(2):52-4, 57-60, 63-4; quiz 66-7. PubMed ID: 15782848
    [TBL] [Abstract][Full Text] [Related]  

  • 11. The discovery of medicines for rare diseases.
    Swinney DC; Xia S
    Future Med Chem; 2014 Jun; 6(9):987-1002. PubMed ID: 25068983
    [TBL] [Abstract][Full Text] [Related]  

  • 12. The role of academic institutions in the development of drugs for rare and neglected diseases.
    Coles LD; Cloyd JC
    Clin Pharmacol Ther; 2012 Aug; 92(2):193-202. PubMed ID: 22760003
    [TBL] [Abstract][Full Text] [Related]  

  • 13. A journey of hope: lessons learned from studies on rare diseases and orphan drugs.
    Wästfelt M; Fadeel B; Henter JI
    J Intern Med; 2006 Jul; 260(1):1-10. PubMed ID: 16789973
    [TBL] [Abstract][Full Text] [Related]  

  • 14. Optimal therapy for rare disorders and genetic diseases: ethical and political challenges.
    MacLeod S
    Proc West Pharmacol Soc; 2007; 50():21-3. PubMed ID: 18605224
    [TBL] [Abstract][Full Text] [Related]  

  • 15. Gene-targeting pharmaceuticals for single-gene disorders.
    Beaudet AL; Meng L
    Hum Mol Genet; 2016 Apr; 25(R1):R18-26. PubMed ID: 26628634
    [TBL] [Abstract][Full Text] [Related]  

  • 16. DIGNiFI: Discovering causative genes for orphan diseases using protein-protein interaction networks.
    Liu X; Yang Z; Lin H; Simmons M; Lu Z
    BMC Syst Biol; 2017 Mar; 11(Suppl 3):23. PubMed ID: 28361678
    [TBL] [Abstract][Full Text] [Related]  

  • 17. The use or generation of biomedical data and existing medicines to discover and establish new treatments for patients with rare diseases - recommendations of the IRDiRC Data Mining and Repurposing Task Force.
    Southall NT; Natarajan M; Lau LPL; Jonker AH; Deprez B; Guilliams T; Hunter L; Rademaker CM; Hivert V; Ardigò D;
    Orphanet J Rare Dis; 2019 Oct; 14(1):225. PubMed ID: 31615551
    [TBL] [Abstract][Full Text] [Related]  

  • 18. 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]  

  • 19. Drugs for rare disorders.
    Cremers S; Aronson JK
    Br J Clin Pharmacol; 2017 Aug; 83(8):1607-1613. PubMed ID: 28653488
    [TBL] [Abstract][Full Text] [Related]  

  • 20. The Orphan Drug Act: Restoring the Mission to Rare Diseases.
    Daniel MG; Pawlik TM; Fader AN; Esnaola NF; Makary MA
    Am J Clin Oncol; 2016 Apr; 39(2):210-3. PubMed ID: 26580246
    [TBL] [Abstract][Full Text] [Related]  

    [Next]    [New Search]
    of 13.