BIOMARKERS

Molecular Biopsy of Human Tumors

- a resource for Precision Medicine *

132 related articles for article (PubMed ID: 31454301)

  • 1. Demonstrating effectiveness or demonstrating not ineffectiveness - A potential solution for rare disease drug product development?
    Chow SC; Huang Z
    J Biopharm Stat; 2019; 29(5):897-907. PubMed ID: 31454301
    [TBL] [Abstract][Full Text] [Related]  

  • 2. 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]  

  • 3. 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]  

  • 4. Probability monitoring procedures for sample size determination.
    Huang Z; Chow SC
    J Biopharm Stat; 2019; 29(5):887-896. PubMed ID: 31454274
    [TBL] [Abstract][Full Text] [Related]  

  • 5. Flexibility in the FDA approach to orphan drug development.
    Hunter NL; Rao GR; Sherman RE
    Nat Rev Drug Discov; 2017 Nov; 16(11):737-738. PubMed ID: 28860647
    [No Abstract]   [Full Text] [Related]  

  • 6. Characteristics of rare disease marketing applications associated with FDA product approvals 2006-2010.
    Pariser AR; Slack DJ; Bauer LJ; Warner CA; Tracy LA
    Drug Discov Today; 2012 Aug; 17(15-16):898-904. PubMed ID: 22564486
    [TBL] [Abstract][Full Text] [Related]  

  • 7. Innovative thinking of clinical investigation for rare disease drug development.
    Wang P; Chow SC
    Orphanet J Rare Dis; 2023 Sep; 18(1):299. PubMed ID: 37740206
    [TBL] [Abstract][Full Text] [Related]  

  • 8. Post-approval Studies for Rare Disease Treatments and Orphan Drugs.
    Maier WC; Christensen RA; Anderson P
    Adv Exp Med Biol; 2017; 1031():197-205. PubMed ID: 29214573
    [TBL] [Abstract][Full Text] [Related]  

  • 9. 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]  

  • 10. Accessing the accelerated approval pathway for rare disease therapeutics.
    Kakkis ED; Kowalcyk S; Bronstein MG
    Nat Biotechnol; 2016 Apr; 34(4):380-3. PubMed ID: 27054988
    [No Abstract]   [Full Text] [Related]  

  • 11. Regulators adopt more orphan drugs.
    Reardon S
    Nature; 2014 Apr; 508(7494):16-7. PubMed ID: 24695293
    [No Abstract]   [Full Text] [Related]  

  • 12. Model-Informed Approach Supporting Drug Development and Regulatory Evaluation for Rare Diseases.
    Li RJ; Ma L; Li F; Li L; Bi Y; Yuan Y; Li Y; Xu Y; Zhang X; Liu J; Bhattaram VA; Wang J; Schuck R; Pacanowski M; Zhu H
    J Clin Pharmacol; 2022 Dec; 62 Suppl 2():S27-S37. PubMed ID: 36461744
    [TBL] [Abstract][Full Text] [Related]  

  • 13. Novel drug targets in 2019.
    Avram S; Halip L; Curpan R; Oprea TI
    Nat Rev Drug Discov; 2020 May; 19(5):300. PubMed ID: 32273590
    [No Abstract]   [Full Text] [Related]  

  • 14. 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]  

  • 15. 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]  

  • 16. Drugs Against Rare Diseases: Are The Regulatory Standards Higher?
    Gobburu J; Pastoor D
    Clin Pharmacol Ther; 2016 Oct; 100(4):322-3. PubMed ID: 27326701
    [TBL] [Abstract][Full Text] [Related]  

  • 17. Assessing the potential clinical impact of reciprocal drug approval legislation on access to novel therapeutics in the USA: a cohort study.
    Larochelle M; Downing NS; Ross JS; David FS
    BMJ Open; 2017 Feb; 7(2):e014582. PubMed ID: 28179418
    [TBL] [Abstract][Full Text] [Related]  

  • 18. Canadian and US drug approval times and safety considerations.
    Rawson NS; Kaitin KI
    Ann Pharmacother; 2003 Oct; 37(10):1403-8. PubMed ID: 14519031
    [TBL] [Abstract][Full Text] [Related]  

  • 19. A personal perspective of orphan drug development for rare diseases: A golden opportunity or an unsustainable future?
    Oo C; Rusch LM
    J Clin Pharmacol; 2016 Mar; 56(3):257-9. PubMed ID: 26211513
    [No Abstract]   [Full Text] [Related]  

  • 20. 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]  

    [Next]    [New Search]
    of 7.