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Volumn 18, Issue 1 SUPPL., 2012, Pages

Clinical Trials In BMT: Ensuring That Rare Diseases and Rarer Therapies are Well Done

Author keywords

[No Author keywords available]

Indexed keywords

ARTICLE; BONE MARROW TRANSPLANTATION; CLINICAL EFFECTIVENESS; CLINICAL TRIAL (TOPIC); FEASIBILITY STUDY; FOLLOW UP; FUNDING; HEALTH CARE COST; HEALTH CARE SYSTEM; HEMATOPOIETIC STEM CELL TRANSPLANTATION; NATIONAL HEALTH ORGANIZATION; OUTCOME ASSESSMENT; POLICY; PRODUCT DEVELOPMENT; QUALITY CONTROL; RARE DISEASE; SAMPLING; STATISTICAL PARAMETERS; STUDY DESIGN; TOXICITY;

EID: 84855393641     PISSN: 10838791     EISSN: 15236536     Source Type: Journal    
DOI: 10.1016/j.bbmt.2011.10.030     Document Type: Article
Times cited : (7)

References (12)
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    • (2001) Small Clinical Trials: Issues and Challenges
  • 2
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    • Clinical trials and rare diseases
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    • Lagakos, S.1
  • 3
    • 0035000904 scopus 로고    scopus 로고
    • Rare diseases and the assessment of intervention: what sorts of clinical trials can we use?
    • Wilcken B. Rare diseases and the assessment of intervention: what sorts of clinical trials can we use?. J Inherit 2001, 24:294-298.
    • (2001) J Inherit , vol.24 , pp. 294-298
    • Wilcken, B.1
  • 4
    • 33947518833 scopus 로고    scopus 로고
    • Challenges in the design and conduct of therapeutic trials in channel disorders
    • Venance S., Herr B.E., Griggs R.C. Challenges in the design and conduct of therapeutic trials in channel disorders. Neurotherapeutics 2007, 4:199-204.
    • (2007) Neurotherapeutics , vol.4 , pp. 199-204
    • Venance, S.1    Herr, B.E.2    Griggs, R.C.3
  • 5
    • 84855387153 scopus 로고    scopus 로고
    • Guidance for Industry: Adaptive Design Clinical Trials for Drugs and Biologics, U.S. Department of Health and Human Services Food and Drug Administration, Center for Drug Evaluation and Research (CDER) Center for Biologics Evaluation and Research (CBER), February
    • Guidance for Industry: Adaptive Design Clinical Trials for Drugs and Biologics, U.S. Department of Health and Human Services Food and Drug Administration, Center for Drug Evaluation and Research (CDER) Center for Biologics Evaluation and Research (CBER), February 2010.
    • (2010)
  • 7
    • 79952060054 scopus 로고    scopus 로고
    • Rare diseases-avoiding misperceptions and establishing realities: the need for reliable epidemiological data
    • Groft S.C., de la Paz M.P. Rare diseases-avoiding misperceptions and establishing realities: the need for reliable epidemiological data. Adv Exp Med Biol 2010, 686:3-14.
    • (2010) Adv Exp Med Biol , vol.686 , pp. 3-14
    • Groft, S.C.1    de la Paz, M.P.2
  • 9
    • 84855374307 scopus 로고    scopus 로고
    • Creating a global rare disease patient registry linked to a rare diseases biorepository database: Rare Disease-HUB (RD-HUB).
    • Rubinstein YR, Groft SC, Bartek R. Creating a global rare disease patient registry linked to a rare diseases biorepository database: Rare Disease-HUB (RD-HUB).
    • Rubinstein, Y.R.1    Groft, S.C.2    Bartek, R.3
  • 10
    • 77957749180 scopus 로고    scopus 로고
    • Contemp Clin Trials.
    • Contemp Clin Trials. 2010;31:394-404.
    • (2010) , vol.31 , pp. 394-404
  • 11
    • 57649109461 scopus 로고    scopus 로고
    • Clinical research for rare disease: opportunities, challenges, and solutions
    • Griggs R.C., Batshaw M., Dunkle M., et al. Clinical research for rare disease: opportunities, challenges, and solutions. Mol Genet Metab 2009, 96:20-26.
    • (2009) Mol Genet Metab , vol.96 , pp. 20-26
    • Griggs, R.C.1    Batshaw, M.2    Dunkle, M.3
  • 12
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    • editors. Rare Diseases and Orphan Products: Accelerating Research and Development. IOM (Institute of Medicine). Washington, DC: The National Academies Press
    • Field MJ, Boat TF, editors. Rare Diseases and Orphan Products: Accelerating Research and Development. IOM (Institute of Medicine). Washington, DC: The National Academies Press, 2010.
    • (2010)
    • Field, M.J.1    Boat, T.F.2


* 이 정보는 Elsevier사의 SCOPUS DB에서 KISTI가 분석하여 추출한 것입니다.