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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
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Cited By
References
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
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
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
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
Prospective Turkish Cohort Study to Investigate the Frequency of Niemann-Pick Disease Type C Mutations in Consanguineous Families with at Least One Homozygous Family Member
Molecular Diagnosis & Therapy
◽
10.1007/s40291-017-0293-9
◽
2017
◽
Vol 21
(6)
◽
pp. 643-651
◽
Cited By ~ 3
Author(s):
Meral Topçu
◽
Dilek Aktas
◽
Merih Öztoprak
◽
Neslihan Önenli Mungan
◽
Aysel Yuce
◽
...
Keyword(s):
Cohort Study
◽
Family Member
◽
Disease Type
◽
Niemann Pick Disease
◽
Type C
◽
Niemann Pick
◽
Pick Disease
Download Full-text
Correction to: Clinical disease progression and biomarkers in Niemann–Pick disease type C: a prospective cohort study
Orphanet Journal of Rare Diseases
◽
10.1186/s13023-021-01855-9
◽
2021
◽
Vol 16
(1)
◽
Author(s):
Eugen Mengel
◽
Bruno Bembi
◽
Mireia del Toro
◽
Federica Deodato
◽
Matthias Gautschi
◽
...
Keyword(s):
Cohort Study
◽
Disease Progression
◽
Prospective Cohort Study
◽
Prospective Cohort
◽
Disease Type
◽
Niemann Pick Disease
◽
Type C
◽
Niemann Pick
◽
Pick Disease
◽
Clinical Disease Progression
An amendment to this paper has been published and can be accessed via the original article.
Download Full-text
Niemann-Pick Disease Type C Quality-of-Life Scale for Adults
PsycTESTS Dataset
◽
10.1037/t75153-000
◽
2019
◽
Author(s):
Lydia Aston
◽
Rachel Shaw
◽
Rebecca Knibb
Keyword(s):
Quality Of Life
◽
Disease Type
◽
Niemann Pick Disease
◽
Quality Of Life Scale
◽
Life Scale
◽
Type C
◽
Niemann Pick
◽
Pick Disease
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ANALYSIS OF CHOLESTEROL AND OXYSTEROL LEVELS IN NIEMANN PICK DISEASE TYPE C MICE AND PATIENTS
Neuropediatrics
◽
10.1055/s-2006-945720
◽
2006
◽
Vol 37
(S 1)
◽
Author(s):
S Tay
◽
X He
◽
AM Jenner
◽
BS Wong
◽
WY Ong
Keyword(s):
Disease Type
◽
Niemann Pick Disease
◽
Type C
◽
Niemann Pick
◽
Pick Disease
Download Full-text
Orphan designation: Hydroxy-propyl-beta-cyclodextrin, Treatment of Niemann-Pick disease, type C
Case Medical Research
◽
10.31525/cmr-399673
◽
2019
◽
Author(s):
Keyword(s):
Disease Type
◽
Orphan Designation
◽
Beta Cyclodextrin
◽
Niemann Pick Disease
◽
Type C
◽
Niemann Pick
◽
Pick Disease
Download Full-text
Orphan designation: miglustat, Treatment of Niemann-Pick disease, type C
Case Medical Research
◽
10.31525/cmr-bc1108
◽
2019
◽
Author(s):
Keyword(s):
Disease Type
◽
Orphan Designation
◽
Niemann Pick Disease
◽
Type C
◽
Niemann Pick
◽
Pick Disease
Download Full-text
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