-
1
-
-
0003916778
-
-
The National Academies Press, Washington, DC, C.H. Evans, S.T. Ilstad (Eds.)
-
Small Clinical Trials: Issues and Challenges 2001, The National Academies Press, Washington, DC. C.H. Evans, S.T. Ilstad (Eds.).
-
(2001)
Small Clinical Trials: Issues and Challenges
-
-
-
2
-
-
0037905488
-
Clinical trials and rare diseases
-
Lagakos S. Clinical trials and rare diseases. N Engl J Med 2003, 348:2455-2456.
-
(2003)
N Engl J Med
, vol.348
, pp. 2455-2456
-
-
Lagakos, S.1
-
3
-
-
0035000904
-
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
-
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
-
-
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)
-
-
-
6
-
-
84855364630
-
-
The National Academies Press, Washington, DC, Available at:, S.N. Nass, H.L. Moses, J. Mendelsohn (Eds.)
-
A National Cancer Clinical Trials System for the 21st Century: Reinvigorating the NCI Cooperative Group Program 2010, The National Academies Press, Washington, DC, Available at:. http://www.nap.edu/catalog/12879.html, S.N. Nass, H.L. Moses, J. Mendelsohn (Eds.).
-
(2010)
A National Cancer Clinical Trials System for the 21st Century: Reinvigorating the NCI Cooperative Group Program
-
-
-
7
-
-
79952060054
-
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
-
8
-
-
79953161288
-
The case for a global rare-diseases registry
-
Forrest C.B., Bartek R.J., Rubinstein Y., Groft S.C. The case for a global rare-diseases registry. Lancet 2011, 377:1057-1059.
-
(2011)
Lancet
, vol.377
, pp. 1057-1059
-
-
Forrest, C.B.1
Bartek, R.J.2
Rubinstein, Y.3
Groft, S.C.4
-
9
-
-
84855374307
-
-
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
-
-
Contemp Clin Trials.
-
Contemp Clin Trials. 2010;31:394-404.
-
(2010)
, vol.31
, pp. 394-404
-
-
-
11
-
-
57649109461
-
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
-
-
84855364628
-
-
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
|