-
2
-
-
70449507812
-
Drug development for orphan diseases in the context of personalized medicine
-
Brewer G. J.Drug development for orphan diseases in the context of personalized medicine.Translational Research. 2009;154:314-322.
-
(2009)
Translational Research
, vol.154
, pp. 314-322
-
-
Brewer, G.J.1
-
3
-
-
38449116688
-
The impact of juvenile Huntington's disease on the family: The case of a rare childhood condition
-
Brewer H. M.,Eatough V.,Smith J. A.,Stanley C. A.,Glendinning N. W.,Quarrell O. W. J.The impact of juvenile Huntington's disease on the family: The case of a rare childhood condition.Journal of Health Psychology. 2008;13 (1): 5-16.
-
(2008)
Journal of Health Psychology
, vol.13
, Issue.1
, pp. 5-16
-
-
Brewer, H.M.1
Eatough, V.2
Smith, J.A.3
Stanley, C.A.4
Glendinning, N.W.5
Quarrell, O.W.J.6
-
6
-
-
33645119030
-
Stress and well-being among parents of children with rare diseases: A prospective intervention study
-
Dellve L.,Samuelsson L.,Tallborn A.,Fasth A.,Hallberg L. R. M.Stress and well-being among parents of children with rare diseases: A prospective intervention study.Journal of Advanced Nursing. 2006;53:392-402.
-
(2006)
Journal of Advanced Nursing
, vol.53
, pp. 392-402
-
-
Dellve, L.1
Samuelsson, L.2
Tallborn, A.3
Fasth, A.4
Hallberg, L.R.M.5
-
8
-
-
57649109461
-
Clinical research for rare disease: Opportunities, challenges, and solutions
-
Griggs R. C.,Batshaw M.,Dunkle M.,Gopal-Srivastava R.,Kaye E.,Krischer J.,Merkel P. A.Clinical research for rare disease: Opportunities, challenges, and solutions.Molecular Genetics and Metabolism. 2009;96 (1): 20-26.
-
(2009)
Molecular Genetics and Metabolism
, vol.96
, Issue.1
, pp. 20-26
-
-
Griggs, R.C.1
Batshaw, M.2
Dunkle, M.3
Gopal-Srivastava, R.4
Kaye, E.5
Krischer, J.6
Merkel, P.A.7
-
10
-
-
70449503966
-
What, if anything, is specific about having a rare disorder? Patients' judgements on being ill and being rare
-
Huyard C.What, if anything, is specific about having a rare disorder? Patients' judgements on being ill and being rare.Health Expectations. 2009;12:361-370.
-
(2009)
Health Expectations
, vol.12
, pp. 361-370
-
-
Huyard, C.1
-
11
-
-
49549108944
-
The role of evidence-based medicine and clinical trials in rare genetic disorders
-
Kruer M. C.,Steiner R. D.The role of evidence-based medicine and clinical trials in rare genetic disorders.Clinical Genetics. 2008;74:197-207.
-
(2008)
Clinical Genetics
, vol.74
, pp. 197-207
-
-
Kruer, M.C.1
Steiner, R.D.2
-
14
-
-
13144272331
-
-
Pub. L
-
Orphan Drug Act, Pub. L. No. 97-414 (1983).
-
(1983)
Orphan Drug Act
, pp. 97-414
-
-
-
15
-
-
42149107453
-
Perceptions of discrimination among persons who have undergone predictive testing for Huntington's disease
-
Penziner E.,Williams J.,Erwin C.,Bombard Y.,Wallis A.,Beglinger L.Paulsen J. S.Perceptions of discrimination among persons who have undergone predictive testing for Huntington's disease.American Journal of Medical Genetics Part B (Neuropsychiatric Genetics), 147B. 2008;:320-325.
-
(2008)
American Journal of Medical Genetics Part B (Neuropsychiatric Genetics), 147B
, pp. 320-325
-
-
Penziner, E.1
Williams, J.2
Erwin, C.3
Bombard, Y.4
Wallis, A.5
Beglinger, L.6
Paulsen, J.S.7
-
16
-
-
77149152241
-
-
Pub. L
-
Rare Diseases Act, Pub. L. No. 107-280 (2002).
-
(2002)
Rare Diseases Act
, pp. 107-280
-
-
-
18
-
-
44849113819
-
Rare diseases: What's next?
-
Remuzzi G.,Garrattini S.Rare diseases: What's next?.Lancet. 2008;371:1978-1979.
-
(2008)
Lancet
, vol.371
, pp. 1978-1979
-
-
Remuzzi, G.1
Garrattini, S.2
-
19
-
-
35748960343
-
Rare diseases, orphan drugs, and orphaned patients
-
Scheindlin S.Rare diseases, orphan drugs, and orphaned patients.Molecular Interventions. 2006;6:186-191.
-
(2006)
Molecular Interventions
, vol.6
, pp. 186-191
-
-
Scheindlin, S.1
-
20
-
-
44849143056
-
Why rare diseases are an important medical and social issue
-
Schieppati A.,Henter J.-I.,Daina E.,Aperia A.Why rare diseases are an important medical and social issue.Lancet. 2008;371:2039-2041.
-
(2008)
Lancet
, vol.371
, pp. 2039-2041
-
-
Schieppati, A.1
Henter, J.-I.2
Daina, E.3
Aperia, A.4
-
21
-
-
67649411650
-
Expensive drugs for rare disorders: To treat or not to treat? The case of enzyme replacement therapy for mucopolysaccharidosis VI
-
Schlander M.,Beck M.Expensive drugs for rare disorders: To treat or not to treat? The case of enzyme replacement therapy for mucopolysaccharidosis VI.Current Medical Research and Opinion. 2009;25:1285-1293.
-
(2009)
Current Medical Research and Opinion
, vol.25
, pp. 1285-1293
-
-
Schlander, M.1
Beck, M.2
-
22
-
-
70449372647
-
Psychosocial aspects of patients with Niemann-Pick disease, type B
-
Shelly L. H.,Wendy P.,Seymour P.Psychosocial aspects of patients with Niemann-Pick disease, type B.American Journal of Medical Genetics Part A, 149A. 2009;:2430-2436.
-
(2009)
American Journal of Medical Genetics Part A, 149A
, pp. 2430-2436
-
-
Shelly, L.H.1
Wendy, P.2
Seymour, P.3
-
23
-
-
80055122385
-
US Senate Orphan Product Development Act of 2002
-
US SenateOrphan Product Development Act of 2002. 2002Public Law No: 107-281.
-
(2002)
Public Law
, pp. 107-281
-
-
-
24
-
-
77149152241
-
US SenateRare Diseases Act of 2002
-
US Senate Rare Diseases Act of 2002. 2002Public Law No: 107-280.
-
(2002)
Public Law
, pp. 107-280
-
-
-
25
-
-
44449152155
-
Developing a national collaborative study system for rare genetic diseases
-
Watson M.,Epstein C.,Howell R. R.,Jones M.,Korf B. R.,McCabe E.,Simpson J. L.Developing a national collaborative study system for rare genetic diseases.Genetics in Medicine. 2008;10:325-329.
-
(2008)
Genetics in Medicine
, vol.10
, pp. 325-329
-
-
Watson, M.1
Epstein, C.2
Howell, R.R.3
Jones, M.4
Korf, B.R.5
McCabe, E.6
Simpson, J.L.7
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