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Natural history of Type A Niemann-Pick disease: Possible endpoints for therapeutic trials
Neurology
◽
10.1212/01.wnl.0000194208.08904.0c
◽
2006
◽
Vol 66
(2)
◽
pp. 228-232
◽
Cited By ~ 68
Author(s):
M. M. McGovern
◽
A. Aron
◽
S. E. Brodie
◽
R. J. Desnick
◽
M. P. Wasserstein
Keyword(s):
Natural History
◽
Type A
◽
Therapeutic Trials
◽
Niemann Pick Disease
◽
History Of
◽
Niemann Pick
◽
Pick Disease
Download Full-text
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Cited By
References
Niemann-Pick Disease Type A and B- Natural History Of Lysosomal Sphingomyelinase
Trafficking of Intracellular Membranes:
◽
10.1007/978-3-642-79547-3_16
◽
1995
◽
pp. 247-263
Author(s):
Klaus Feriinz
◽
Konrad Sandhoff
Keyword(s):
Natural History
◽
Type A
◽
Disease Type
◽
Niemann Pick Disease
◽
History Of
◽
Niemann Pick
◽
Pick Disease
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Natural history of Niemann-Pick disease type C in a multicentre observational retrospective cohort study
Molecular Genetics and Metabolism
◽
10.1016/j.ymgme.2009.06.009
◽
2009
◽
Vol 98
(3)
◽
pp. 250-254
◽
Cited By ~ 66
Author(s):
J.E. Wraith
◽
N. Guffon
◽
M. Rohrbach
◽
W.L. Hwu
◽
G.C. Korenke
◽
...
Keyword(s):
Cohort Study
◽
Natural History
◽
Retrospective Cohort Study
◽
Retrospective Cohort
◽
Disease Type
◽
Niemann Pick Disease
◽
History Of
◽
Type C
◽
Niemann Pick
◽
Pick Disease
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A Prospective, Cross-sectional Survey Study of the Natural History of Niemann-Pick Disease Type B
PEDIATRICS
◽
10.1542/peds.2007-3016
◽
2008
◽
Vol 122
(2)
◽
pp. e341-e349
◽
Cited By ~ 92
Author(s):
M. M. McGovern
◽
M. P. Wasserstein
◽
R. Giugliani
◽
B. Bembi
◽
M. T. Vanier
◽
...
Keyword(s):
Natural History
◽
Disease Type
◽
Survey Study
◽
Type B
◽
Cross Sectional Survey
◽
Cross Sectional
◽
Niemann Pick Disease
◽
History Of
◽
Niemann Pick
◽
Pick Disease
Download Full-text
46. The natural history of Niemann–Pick disease type C
Molecular Genetics and Metabolism
◽
10.1016/j.ymgme.2007.10.058
◽
2008
◽
Vol 93
(2)
◽
pp. 25
Author(s):
Elizabeth Jacklin
◽
Jackie Imrie
◽
Suprana Dasgupta
◽
Guy Besley
◽
Harris Chris
◽
...
Keyword(s):
Natural History
◽
Disease Type
◽
Niemann Pick Disease
◽
History Of
◽
Type C
◽
Niemann Pick
◽
Pick Disease
Download Full-text
The Natural History of Type B Niemann-Pick Disease: Results From a 10-Year Longitudinal Study
PEDIATRICS
◽
10.1542/peds.2004-0887
◽
2004
◽
Vol 114
(6)
◽
pp. e672-e677
◽
Cited By ~ 72
Author(s):
M. P. Wasserstein
◽
R. J. Desnick
◽
E. H. Schuchman
◽
S. Hossain
◽
S. Wallenstein
◽
...
Keyword(s):
Longitudinal Study
◽
Natural History
◽
Type B
◽
Niemann Pick Disease
◽
History Of
◽
Niemann Pick
◽
Pick Disease
Download Full-text
P304 Natural history of Niemann Pick disease type C in a multicentre observational retrospective cohort study
European Journal of Paediatric Neurology
◽
10.1016/s1090-3798(09)70362-9
◽
2009
◽
Vol 13
◽
pp. S115-S116
Author(s):
J.E. Wraith
◽
N. Guffon
◽
M. Rohrbach
◽
W.-L. Hwu
◽
C. Korenke
◽
...
Keyword(s):
Cohort Study
◽
Natural History
◽
Retrospective Cohort Study
◽
Retrospective Cohort
◽
Disease Type
◽
Niemann Pick Disease
◽
History Of
◽
Type C
◽
Niemann Pick
◽
Pick Disease
Download Full-text
The natural history of Niemann–Pick disease type C in the UK
Journal of Inherited Metabolic Disease
◽
10.1007/s10545-006-0384-7
◽
2006
◽
Vol 30
(1)
◽
pp. 51-59
◽
Cited By ~ 97
Author(s):
J. Imrie
◽
S. Dasgupta
◽
G. T. N. Besley
◽
C. Harris
◽
L. Heptinstall
◽
...
Keyword(s):
Natural History
◽
Disease Type
◽
Niemann Pick Disease
◽
History Of
◽
Type C
◽
Niemann Pick
◽
The Uk
◽
Pick Disease
Download Full-text
The natural history of Niemann–Pick disease type C in the UK
Journal of Inherited Metabolic Disease
◽
10.1007/s10545-007-9986-2
◽
2007
◽
Vol 30
(5)
◽
pp. 833-833
◽
Cited By ~ 2
Author(s):
J. Imrie
◽
S. Dasgupta
◽
G. T. N. Besley
◽
C. Harris
◽
L. Heptinstall
◽
...
Keyword(s):
Natural History
◽
Disease Type
◽
Niemann Pick Disease
◽
History Of
◽
Type C
◽
Niemann Pick
◽
The Uk
◽
Pick Disease
Download Full-text
Signaling pathway for radiation-induced apoptosis in the lymphoblasts from neuronopathic (type A) and non-neuronopathic (type B) forms of Niemann–Pick disease
Journal of the Neurological Sciences
◽
10.1016/s0022-510x(02)00106-5
◽
2002
◽
Vol 199
(1-2)
◽
pp. 39-43
◽
Cited By ~ 3
Author(s):
Tamaki Mikami
◽
Tsutomu Takahashi
◽
Akira Ishida
◽
Yoshihiro Minamiya
◽
Hiroyuki Ida
◽
...
Keyword(s):
Signaling Pathway
◽
Type A
◽
Type B
◽
Niemann Pick Disease
◽
Radiation Induced
◽
Niemann Pick
◽
Induced Apoptosis
◽
Pick Disease
Download Full-text
Observational cohort study of the natural history of Niemann-Pick disease type C in the UK: a 5-year update from the UK clinical database
BMC Neurology
◽
10.1186/s12883-015-0511-1
◽
2015
◽
Vol 15
(1)
◽
Cited By ~ 40
Author(s):
Jackie Imrie
◽
Lesley Heptinstall
◽
Stephen Knight
◽
Kate Strong
Keyword(s):
Disease Type
◽
Observational Cohort Study
◽
Clinical Database
◽
Niemann Pick Disease
◽
History Of
◽
Observational Cohort
◽
Type C
◽
Niemann Pick
◽
The Uk
◽
Pick Disease
Download Full-text
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