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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
Download Full-text
Related Documents
Cited By
References
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
P302 A multicentre retrospective cohort study of miglustat in patients with Niemann Pick disease type C
European Journal of Paediatric Neurology
◽
10.1016/s1090-3798(09)70360-5
◽
2009
◽
Vol 13
◽
pp. S115
Author(s):
M. Pineda
◽
J.E. Wraith
◽
E. Mengel
◽
F. Sedel
◽
W.-L. Hwu
◽
...
Keyword(s):
Cohort Study
◽
Retrospective Cohort Study
◽
Retrospective Cohort
◽
Disease Type
◽
Niemann Pick Disease
◽
Type C
◽
Niemann Pick
◽
Pick Disease
Download Full-text
Miglustat in patients with Niemann-Pick disease Type C (NP-C): A multicenter observational retrospective cohort study
Molecular Genetics and Metabolism
◽
10.1016/j.ymgme.2009.07.003
◽
2009
◽
Vol 98
(3)
◽
pp. 243-249
◽
Cited By ~ 126
Author(s):
M. Pineda
◽
J.E. Wraith
◽
E. Mengel
◽
F. Sedel
◽
W.-L. Hwu
◽
...
Keyword(s):
Cohort Study
◽
Retrospective Cohort Study
◽
Retrospective Cohort
◽
Disease Type
◽
Niemann Pick Disease
◽
Type C
◽
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 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
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
Evaluation of age of death in Niemann-Pick disease, type C: Utility of disease support group websites to understand natural history
Molecular Genetics and Metabolism
◽
10.1016/j.ymgme.2019.02.004
◽
2019
◽
Vol 126
(4)
◽
pp. 466-469
◽
Cited By ~ 7
Author(s):
Simona E. Bianconi
◽
Dylan I. Hammond
◽
Nicole Y. Farhat
◽
An Dang Do
◽
Kisha Jenkins
◽
...
Keyword(s):
Natural History
◽
Support Group
◽
Disease Type
◽
Niemann Pick Disease
◽
Type C
◽
Niemann Pick
◽
Pick Disease
◽
Age Of Death
Download Full-text
Stable or improved neurological manifestations during miglustat therapy in patients from the international disease registry for Niemann-Pick disease type C: an observational cohort study
Orphanet Journal of Rare Diseases
◽
10.1186/s13023-015-0284-z
◽
2015
◽
Vol 10
(1)
◽
Cited By ~ 58
Author(s):
Marc C Patterson
◽
◽
Eugen Mengel
◽
Marie T Vanier
◽
Barbara Schwierin
◽
...
Keyword(s):
Cohort Study
◽
Disease Type
◽
Observational Cohort Study
◽
Neurological Manifestations
◽
Disease Registry
◽
Niemann Pick Disease
◽
Observational Cohort
◽
Type C
◽
Niemann Pick
◽
Pick Disease
Download Full-text
Defining Natural History: Assessment of the Ability of College Students to Aid in Characterizing Clinical Progression of Niemann-Pick Disease, Type C
PLoS ONE
◽
10.1371/journal.pone.0023666
◽
2011
◽
Vol 6
(10)
◽
pp. e23666
◽
Cited By ~ 3
Author(s):
Jenny Shin
◽
Katrina Epperson
◽
Nicole M. Yanjanin
◽
Jennifer Albus
◽
Laura Borgenheimer
◽
...
Keyword(s):
College Students
◽
Natural History
◽
Disease Type
◽
Clinical Progression
◽
Niemann Pick Disease
◽
Type C
◽
Niemann Pick
◽
Pick Disease
Download Full-text
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