scholarly journals Long‐term survival outcomes of patients with Niemann‐Pick disease type C receiving miglustat treatment: A large retrospective observational study

2020 ◽  
Vol 43 (5) ◽  
pp. 1060-1069 ◽  
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

scholarly journals Long-term efficacy of miglustat in paediatric patients with Niemann-Pick disease type C

2012 ◽  
Vol 36 (1) ◽  
pp. 129-137 ◽  
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

99. Long-term clinical trial with miglustat in Niemann-Pick disease type C

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

scholarly journals Adult Niemann-Pick disease type C in France: clinical phenotypes and long-term miglustat treatment effect

2018 ◽  
Vol 13 (1) ◽  
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

Neuropsychological and electrophysiological long-term follow-up in the adult form of Niemann-Pick disease type C

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

Long-Term Miglustat Therapy in Children With Niemann-Pick Disease Type C

2009 ◽  
Vol 25 (3) ◽  
pp. 300-305 ◽  
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

scholarly journals 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 ◽  
2019 ◽  
Vol 20 (11) ◽  
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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