BIOMARKERS

Molecular Biopsy of Human Tumors

- a resource for Precision Medicine *

475 related articles for article (PubMed ID: 31836861)

  • 1. Therapies for rare diseases: therapeutic modalities, progress and challenges ahead.
    Tambuyzer E; Vandendriessche B; Austin CP; Brooks PJ; Larsson K; Miller Needleman KI; Valentine J; Davies K; Groft SC; Preti R; Oprea TI; Prunotto M
    Nat Rev Drug Discov; 2020 Feb; 19(2):93-111. PubMed ID: 31836861
    [TBL] [Abstract][Full Text] [Related]  

  • 2. Development of orphan drugs for rare diseases.
    Yoo HW
    Clin Exp Pediatr; 2024 Jul; 67(7):315-327. PubMed ID: 37402468
    [TBL] [Abstract][Full Text] [Related]  

  • 3. Orphan drug designation in Europe: A booster for the research and development of drugs in rare diseases.
    Micallef J; Blin O
    Therapie; 2020 Apr; 75(2):133-139. PubMed ID: 32156423
    [TBL] [Abstract][Full Text] [Related]  

  • 4. 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]  

  • 5. 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]  

  • 6. Pediatric Orphan Drug Indications: 2010-2018.
    Kimmel L; Conti RM; Volerman A; Chua KP
    Pediatrics; 2020 Apr; 145(4):. PubMed ID: 32127360
    [TBL] [Abstract][Full Text] [Related]  

  • 7. Orphan drug development in China: progress and challenges.
    Zhang S; Chen L; Zhang Z; Zhao Y
    Lancet; 2019 Sep; 394(10204):1127-1128. PubMed ID: 31571591
    [No Abstract]   [Full Text] [Related]  

  • 8. How Far Have We Come? Challenges to Orphan Drug Access in China, 2011-2017.
    Guan X; Zhang J; Man C; Ni B; Shi L
    J Pharm Sci; 2019 Jun; 108(6):2199-2205. PubMed ID: 30677418
    [TBL] [Abstract][Full Text] [Related]  

  • 9. Drug Repurposing for Rare Diseases.
    Roessler HI; Knoers NVAM; van Haelst MM; van Haaften G
    Trends Pharmacol Sci; 2021 Apr; 42(4):255-267. PubMed ID: 33563480
    [TBL] [Abstract][Full Text] [Related]  

  • 10. Protein replacement therapies for rare diseases: a breeze for regulatory approval?
    Gorzelany JA; de Souza MP
    Sci Transl Med; 2013 Mar; 5(178):178fs10. PubMed ID: 23536010
    [TBL] [Abstract][Full Text] [Related]  

  • 11. Drug repurposing in rare diseases: Myths and reality.
    Fetro C; Scherman D
    Therapie; 2020 Apr; 75(2):157-160. PubMed ID: 32241561
    [TBL] [Abstract][Full Text] [Related]  

  • 12. Statistical considerations for rare diseases drug development.
    Chow SC; Chang YW
    J Biopharm Stat; 2019; 29(5):874-886. PubMed ID: 31454299
    [TBL] [Abstract][Full Text] [Related]  

  • 13. Novel treatments for rare rheumatologic disorders: analysis of the impact of 30 years of the US orphan drug act.
    Lutz T; Lampert A; Hoffmann GF; Ries M
    Orphanet J Rare Dis; 2016 May; 11(1):60. PubMed ID: 27176041
    [TBL] [Abstract][Full Text] [Related]  

  • 14. Innovative design and analysis for rare disease drug development.
    Chow SC; Huang Z
    J Biopharm Stat; 2020 May; 30(3):537-549. PubMed ID: 32065047
    [TBL] [Abstract][Full Text] [Related]  

  • 15. Orphan drugs, orphan diseases. The first decade of orphan drug legislation in the EU.
    Joppi R; Bertele' V; Garattini S
    Eur J Clin Pharmacol; 2013 Apr; 69(4):1009-24. PubMed ID: 23090701
    [TBL] [Abstract][Full Text] [Related]  

  • 16. Survey of Japanese Orphan Drug Program: Factors Related to Successful Marketing Approval.
    Harada K; Toriyabe K; Ono S
    J Clin Pharmacol; 2020 Jan; 60(1):117-124. PubMed ID: 31364772
    [TBL] [Abstract][Full Text] [Related]  

  • 17. Rare diseases, orphan drugs and their regulation: questions and misconceptions.
    Tambuyzer E
    Nat Rev Drug Discov; 2010 Dec; 9(12):921-9. PubMed ID: 21060315
    [TBL] [Abstract][Full Text] [Related]  

  • 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; 21(4):487-93. PubMed ID: 27022038
    [TBL] [Abstract][Full Text] [Related]  

  • 19. Pressure for drug development in lysosomal storage disorders - a quantitative analysis thirty years beyond the US orphan drug act.
    Mechler K; Mountford WK; Hoffmann GF; Ries M
    Orphanet J Rare Dis; 2015 Apr; 10():46. PubMed ID: 25896727
    [TBL] [Abstract][Full Text] [Related]  

  • 20. Analysis of Incentive Policies and Initiatives on Orphan Drug Development in China: Challenges, Reforms and Implications.
    Zhao Z; Pei Z; Hu A; Zhang Y; Chen J
    Orphanet J Rare Dis; 2023 Jul; 18(1):220. PubMed ID: 37501126
    [TBL] [Abstract][Full Text] [Related]  

    [Next]    [New Search]
    of 24.