BIOMARKERS

Molecular Biopsy of Human Tumors

- a resource for Precision Medicine *

242 related articles for article (PubMed ID: 28815894)

  • 1. Challenges of developing and conducting clinical trials in rare disorders.
    Kempf L; Goldsmith JC; Temple R
    Am J Med Genet A; 2018 Apr; 176(4):773-783. PubMed ID: 28815894
    [TBL] [Abstract][Full Text] [Related]  

  • 2. The future of Cochrane Neonatal.
    Soll RF; Ovelman C; McGuire W
    Early Hum Dev; 2020 Nov; 150():105191. PubMed ID: 33036834
    [TBL] [Abstract][Full Text] [Related]  

  • 3. Patient-Reported Outcome and Observer-Reported Outcome Assessment in Rare Disease Clinical Trials: An ISPOR COA Emerging Good Practices Task Force Report.
    Benjamin K; Vernon MK; Patrick DL; Perfetto E; Nestler-Parr S; Burke L
    Value Health; 2017; 20(7):838-855. PubMed ID: 28712612
    [TBL] [Abstract][Full Text] [Related]  

  • 4. Recommendations for the design of small population clinical trials.
    Day S; Jonker AH; Lau LPL; Hilgers RD; Irony I; Larsson K; Roes KC; Stallard N
    Orphanet J Rare Dis; 2018 Nov; 13(1):195. PubMed ID: 30400970
    [TBL] [Abstract][Full Text] [Related]  

  • 5. Folic acid supplementation and malaria susceptibility and severity among people taking antifolate antimalarial drugs in endemic areas.
    Crider K; Williams J; Qi YP; Gutman J; Yeung L; Mai C; Finkelstain J; Mehta S; Pons-Duran C; Menéndez C; Moraleda C; Rogers L; Daniels K; Green P
    Cochrane Database Syst Rev; 2022 Feb; 2(2022):. PubMed ID: 36321557
    [TBL] [Abstract][Full Text] [Related]  

  • 6. Rare cancer trial design: lessons from FDA approvals.
    Gaddipati H; Liu K; Pariser A; Pazdur R
    Clin Cancer Res; 2012 Oct; 18(19):5172-8. PubMed ID: 22718862
    [TBL] [Abstract][Full Text] [Related]  

  • 7. 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]  

  • 8. Regulatory strategies for rare diseases under current global regulatory statutes: a discussion with stakeholders.
    Mulberg AE; Bucci-Rechtweg C; Giuliano J; Jacoby D; Johnson FK; Liu Q; Marsden D; McGoohan S; Nelson R; Patel N; Romero K; Sinha V; Sitaraman S; Spaltro J; Kessler V
    Orphanet J Rare Dis; 2019 Feb; 14(1):36. PubMed ID: 30736861
    [TBL] [Abstract][Full Text] [Related]  

  • 9. Dose-finding studies in drug development for rare genetic diseases.
    Wang L; Wang J; Feng J; Doi M; Pepe S; Pacanowski M; Schuck RN
    Orphanet J Rare Dis; 2022 Apr; 17(1):156. PubMed ID: 35382851
    [TBL] [Abstract][Full Text] [Related]  

  • 10. Navigating the Regulatory Landscape to Develop Pediatric Oncology Drugs: Expert Opinion Recommendations.
    Barry E; Walsh JA; Weinrich SL; Beaupre D; Blasi E; Arenson DR; Jacobs IA
    Paediatr Drugs; 2021 Jul; 23(4):381-394. PubMed ID: 34173206
    [TBL] [Abstract][Full Text] [Related]  

  • 11. [The stakes of pre- and post-market authorization development and clinical assessment].
    Schwebig A
    Presse Med; 2012 May; 41 Suppl 1():S6-8. PubMed ID: 22483770
    [No Abstract]   [Full Text] [Related]  

  • 12. Strategies for postmarketing surveillance of drugs for rare diseases.
    Kesselheim AS; Gagne JJ
    Clin Pharmacol Ther; 2014 Mar; 95(3):265-8. PubMed ID: 24193169
    [TBL] [Abstract][Full Text] [Related]  

  • 13. Preparing for the Future of Rare Diseases.
    Groft SC; Posada de la Paz M
    Adv Exp Med Biol; 2017; 1031():641-648. PubMed ID: 29214596
    [TBL] [Abstract][Full Text] [Related]  

  • 14. Effective Market Exclusivity of New Molecular Entities for Rare and Non-rare Diseases.
    Kerr KW; Glos LJ
    Pharmaceut Med; 2020 Feb; 34(1):19-29. PubMed ID: 32048213
    [TBL] [Abstract][Full Text] [Related]  

  • 15. FDA orphan products clinical trial grants: assessment of outcomes and impact on rare disease product development.
    Miller KL; Mueller C; Liu G; Miller Needleman KI; Maynard J
    Orphanet J Rare Dis; 2020 Sep; 15(1):234. PubMed ID: 32883327
    [TBL] [Abstract][Full Text] [Related]  

  • 16. Characteristics of non-randomised studies using comparisons with external controls submitted for regulatory approval in the USA and Europe: a systematic review.
    Goring S; Taylor A; Müller K; Li TJJ; Korol EE; Levy AR; Freemantle N
    BMJ Open; 2019 Feb; 9(2):e024895. PubMed ID: 30819708
    [TBL] [Abstract][Full Text] [Related]  

  • 17. 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]  

  • 18. Incentives for orphan drug research and development in the United States.
    Seoane-Vazquez E; Rodriguez-Monguio R; Szeinbach SL; Visaria J
    Orphanet J Rare Dis; 2008 Dec; 3():33. PubMed ID: 19087348
    [TBL] [Abstract][Full Text] [Related]  

  • 19. Need to improve clinical trials in rare neurodegenerative disorders.
    Puopolo M; Pocchiari M
    Ann Ist Super Sanita; 2011; 47(1):55-9. PubMed ID: 21430340
    [TBL] [Abstract][Full Text] [Related]  

  • 20. A checklist for clinical trials in rare disease: obstacles and anticipatory actions-lessons learned from the FOR-DMD trial.
    Crow RA; Hart KA; McDermott MP; Tawil R; Martens WB; Herr BE; McColl E; Wilkinson J; Kirschner J; King WM; Eagle M; Brown MW; Hirtz D; Lochmuller H; Straub V; Ciafaloni E; Shieh PB; Spinty S; Childs AM; Manzur AY; Morandi L; Butterfield RJ; Horrocks I; Roper H; Flanigan KM; Kuntz NL; Mah JK; Morrison L; Darras BT; von der Hagen M; Schara U; Wilichowski E; Mongini T; McDonald CM; Vita G; Barohn RJ; Finkel RS; Wicklund M; McMillan HJ; Hughes I; Pegoraro E; Bryan Burnette W; Howard JF; Thangarajh M; Campbell C; Griggs RC; Bushby K; Guglieri M
    Trials; 2018 May; 19(1):291. PubMed ID: 29793540
    [TBL] [Abstract][Full Text] [Related]  

    [Next]    [New Search]
    of 13.