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99. Long-term clinical trial with miglustat in Niemann-Pick disease type C
Molecular Genetics and Metabolism
◽
10.1016/j.ymgme.2008.11.100
◽
2009
◽
Vol 96
(2)
◽
pp. S33-S34
Author(s):
Marc Patterson
◽
Darlenn Vecchio
◽
Elizabeth Jacklin
◽
Ed Wraith
Keyword(s):
Clinical Trial
◽
Disease Type
◽
Niemann Pick Disease
◽
Type C
◽
Niemann Pick
◽
Pick Disease
Download Full-text
Related Documents
Cited By
References
Miglustat in adult and juvenile patients with Niemann–Pick disease type C: Long-term data from a clinical trial
Molecular Genetics and Metabolism
◽
10.1016/j.ymgme.2009.12.006
◽
2010
◽
Vol 99
(4)
◽
pp. 351-357
◽
Cited By ~ 130
Author(s):
James E. Wraith
◽
Darleen Vecchio
◽
Elizabeth Jacklin
◽
Larry Abel
◽
Harbajan Chadha-Boreham
◽
...
Keyword(s):
Clinical Trial
◽
Disease Type
◽
Niemann Pick Disease
◽
Type C
◽
Niemann Pick
◽
Pick Disease
◽
Term Data
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Long-term efficacy of miglustat in paediatric patients with Niemann-Pick disease type C
Journal of Inherited Metabolic Disease
◽
10.1007/s10545-012-9479-9
◽
2012
◽
Vol 36
(1)
◽
pp. 129-137
◽
Cited By ~ 27
Author(s):
Y. H. Chien
◽
S. F. Peng
◽
C. C. Yang
◽
N. C. Lee
◽
L. K. Tsai
◽
...
Keyword(s):
Disease Type
◽
Term Efficacy
◽
Paediatric Patients
◽
Niemann Pick Disease
◽
Type C
◽
Niemann Pick
◽
Pick Disease
Download Full-text
Adult Niemann-Pick disease type C in France: clinical phenotypes and long-term miglustat treatment effect
Orphanet Journal of Rare Diseases
◽
10.1186/s13023-018-0913-4
◽
2018
◽
Vol 13
(1)
◽
Cited By ~ 16
Author(s):
Yann Nadjar
◽
Ana Lucia Hütter-Moncada
◽
Philippe Latour
◽
Xavier Ayrignac
◽
Elsa Kaphan
◽
...
Keyword(s):
Treatment Effect
◽
Disease Type
◽
Clinical Phenotypes
◽
Niemann Pick Disease
◽
Type C
◽
Niemann Pick
◽
Pick Disease
Download Full-text
Neuropsychological and electrophysiological long-term follow-up in the adult form of Niemann-Pick disease type C
Clinical Neurophysiology
◽
10.1016/j.clinph.2018.09.083
◽
2019
◽
Vol 130
(1)
◽
pp. e13-e14
Author(s):
S. Tozza
◽
R. Dubbioso
◽
R. Iodice
◽
A. Topa
◽
M. Esposito
◽
...
Keyword(s):
Disease Type
◽
Adult Form
◽
Niemann Pick Disease
◽
Long Term Follow Up
◽
Type C
◽
Niemann Pick
◽
Pick Disease
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Long-Term Miglustat Therapy in Children With Niemann-Pick Disease Type C
Journal of Child Neurology
◽
10.1177/0883073809344222
◽
2009
◽
Vol 25
(3)
◽
pp. 300-305
◽
Cited By ~ 102
Author(s):
Marc C. Patterson
◽
Darleen Vecchio
◽
Elizabeth Jacklin
◽
Larry Abel
◽
Harbajan Chadha-Boreham
◽
...
Keyword(s):
Disease Type
◽
Niemann Pick Disease
◽
Type C
◽
Niemann Pick
◽
Pick Disease
Download Full-text
NPC 1 enables cholesterol mobilization during long‐term potentiation that can be restored in Niemann–Pick disease type C by CYP 46A1 activation
EMBO Reports
◽
10.15252/embr.201948143
◽
2019
◽
Vol 20
(11)
◽
Cited By ~ 3
Author(s):
Daniel N Mitroi
◽
Guadalupe Pereyra‐Gómez
◽
Beatriz Soto‐Huelin
◽
Fernando Senovilla
◽
Toshihide Kobayashi
◽
...
Keyword(s):
Long Term Potentiation
◽
Disease Type
◽
Niemann Pick Disease
◽
Term Potentiation
◽
Type C
◽
Niemann Pick
◽
Pick Disease
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Preliminary Results on Long-Term Potentiation-Like Cortical Plasticity and Cholinergic Dysfunction After Miglustat Treatment in Niemann-Pick Disease Type C
JIMD Reports - JIMD Reports, Volume 36
◽
10.1007/8904_2016_33
◽
2017
◽
pp. 19-27
◽
Cited By ~ 13
Author(s):
Alberto Benussi
◽
Maria Sofia Cotelli
◽
Maura Cosseddu
◽
Valeria Bertasi
◽
Marinella Turla
◽
...
Keyword(s):
Cortical Plasticity
◽
Long Term Potentiation
◽
Disease Type
◽
Cholinergic Dysfunction
◽
Niemann Pick Disease
◽
Term Potentiation
◽
Type C
◽
Niemann Pick
◽
Pick Disease
Download Full-text
Long‐term survival outcomes of patients with Niemann‐Pick disease type C receiving miglustat treatment: A large retrospective observational study
Journal of Inherited Metabolic Disease
◽
10.1002/jimd.12245
◽
2020
◽
Vol 43
(5)
◽
pp. 1060-1069
◽
Cited By ~ 2
Author(s):
Marc C. Patterson
◽
William S. Garver
◽
Robert Giugliani
◽
Jackie Imrie
◽
Helena Jahnova
◽
...
Keyword(s):
Observational Study
◽
Disease Type
◽
Retrospective Observational Study
◽
Survival Outcomes
◽
Term Survival
◽
Niemann Pick Disease
◽
Type C
◽
Niemann Pick
◽
Pick Disease
Download Full-text
Open‐Label Study of Long‐Term Safety and Efficacy of Intravenous Trappsol Cyclo (HPβCD) in Niemann‐Pick Disease Type C
Case Medical Research
◽
10.31525/ct1-nct03893071
◽
2019
◽
Author(s):
Keyword(s):
Disease Type
◽
Open Label
◽
Open Label Study
◽
Label Study
◽
Safety And Efficacy
◽
Niemann Pick Disease
◽
Type C
◽
Niemann Pick
◽
Pick Disease
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Faculty Opinions recommendation of Long term follow-up to evaluate the efficacy of miglustat treatment in Italian patients with Niemann-Pick disease type C.
Faculty Opinions – Post-Publication Peer Review of the Biomedical Literature
◽
10.3410/f.727681014.793542547
◽
2018
◽
Author(s):
Barbara Borroni
Keyword(s):
Disease Type
◽
Niemann Pick Disease
◽
Long Term Follow Up
◽
Type C
◽
Niemann Pick
◽
Pick Disease
Download Full-text
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