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Journal Abstract Search

416 related items for PubMed ID: 20566615

  • 1. 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
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  • 2. 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
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  • 3. 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
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  • 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
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  • 11. 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
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  • 12. 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 03; 12(1):64. PubMed ID: 28372595
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  • 13. 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 03; 11(59):367-75. PubMed ID: 21524390
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  • 15. Special FDA designations for drug development: orphan, fast track, accelerated approval, priority review, and breakthrough therapy.
    Michaeli DT, Michaeli T, Albers S, Boch T, Michaeli JC.
    Eur J Health Econ; 2024 Aug 03; 25(6):979-997. PubMed ID: 37962724
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  • 16. Investigating the landscape of US orphan product approvals.
    Miller KL, Lanthier M.
    Orphanet J Rare Dis; 2018 Oct 22; 13(1):183. PubMed ID: 30348193
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  • 17. Innovating by developing new uses of already-approved drugs: trends in the marketing approval of supplemental indications.
    DiMasi JA.
    Clin Ther; 2013 Jun 22; 35(6):808-18. PubMed ID: 23726388
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  • 18. 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 22; 21(4):487-93. PubMed ID: 27022038
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  • 19. Despite surge in orphan drug designations, approvals still lag.
    Heger M.
    Nat Med; 2011 Mar 22; 17(3):236. PubMed ID: 21383708
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  • 20. FDA orphan drug designations for lysosomal storage disorders - a cross-sectional analysis.
    Garbade SF, Zielonka M, Mechler K, Kölker S, Hoffmann GF, Staufner C, Mengel E, Ries M.
    PLoS One; 2020 Mar 22; 15(4):e0230898. PubMed ID: 32267884
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