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Journal Abstract Search

422 related items for PubMed ID: 28209180

  • 1. Are products with an orphan designation for oncology indications different from products for other rare indications? A retrospective analysis of European orphan designations granted between 2002-2012.
    Pauwels K, Huys I, Casteels M, Larsson K, Voltz C, Penttila K, Morel T, Simoens S.
    Orphanet J Rare Dis; 2017 Feb 16; 12(1):36. PubMed ID: 28209180
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  • 2. 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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  • 5. [European incentives for orphan medicinal products].
    Enzmann H, Lütz J.
    Bundesgesundheitsblatt Gesundheitsforschung Gesundheitsschutz; 2008 May 03; 51(5):500-8. PubMed ID: 18696141
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  • 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 03; 21(4):487-93. PubMed ID: 27022038
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  • 10. EU orphan regulation--ten years of application.
    Michaux G.
    Food Drug Law J; 2010 Apr 03; 65(4):639-69, i-ii. PubMed ID: 24479246
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  • 12. Orphan drug designation in Europe: A booster for the research and development of drugs in rare diseases.
    Micallef J, Blin O.
    Therapie; 2020 Apr 03; 75(2):133-139. PubMed ID: 32156423
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  • 13. Advancing rare disease treatment: EMA's decade-long insights into engineered adoptive cell therapy for rare cancers and orphan designation.
    Kalland ME, Pose-Boirazian T, Palomo GM, Naumann-Winter F, Costa E, Matusevicius D, Duarte DM, Malikova E, Vitezic D, Larsson K, Magrelli A, Stoyanova-Beninska V, Mariz S.
    Gene Ther; 2024 Jul 03; 31(7-8):366-377. PubMed ID: 38480914
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  • 17. A comprehensive study of the rare diseases and conditions targeted by orphan drug designations and approvals over the forty years of the Orphan Drug Act.
    Fermaglich LJ, Miller KL.
    Orphanet J Rare Dis; 2023 Jun 23; 18(1):163. PubMed ID: 37353796
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  • 19. Analysis of the first ten years of FDA's rare pediatric disease priority review voucher program: designations, diseases, and drug development.
    Mease C, Miller KL, Fermaglich LJ, Best J, Liu G, Torjusen E.
    Orphanet J Rare Dis; 2024 Feb 25; 19(1):86. PubMed ID: 38403586
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  • 20. Drug development for exceptionally rare metabolic diseases: challenging but not impossible.
    Putzeist M, Mantel-Teeuwisse AK, Wied CC, Hoes AW, Leufkens HG, de Vrueh RL.
    Orphanet J Rare Dis; 2013 Nov 15; 8():179. PubMed ID: 24237580
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