A therapy with miglustat, 2-hydroxypropyl-ß-cyclodextrin and allopregnanolone restores splenic cholesterol homeostasis in Niemann-pick disease type C1

Anna-Maria Neßlauer; Anne Gläser; Markus Gräler; Robby Engelmann; Brigitte Müller-Hilke; Marcus Frank; Christine Burstein; Arndt Rolfs; John Neidhardt; Andreas Wree; Martin Witt; Anja U. Bräuer

doi:10.1186/s12944-019-1088-2

A therapy with miglustat, 2-hydroxypropyl-ß-cyclodextrin and allopregnanolone restores splenic cholesterol homeostasis in Niemann-pick disease type C1

Lipids in Health and Disease ◽

10.1186/s12944-019-1088-2 ◽

2019 ◽

Vol 18 (1) ◽

Cited By ~ 6

Author(s):

Anna-Maria Neßlauer ◽

Anne Gläser ◽

Markus Gräler ◽

Robby Engelmann ◽

Brigitte Müller-Hilke ◽

...

Keyword(s):

Disease Type ◽

Cholesterol Homeostasis ◽

Niemann Pick Disease ◽

Niemann Pick ◽

Pick Disease

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Characterization of cholesterol homeostasis in sphingosine-1-phosphate lyase-deficient fibroblasts reveals a Niemann-Pick disease type C-like phenotype with enhanced lysosomal Ca2+ storage

Scientific Reports ◽

10.1038/srep43575 ◽

2017 ◽

Vol 7 (1) ◽

Cited By ~ 7

Author(s):

Hans Vienken ◽

Nathalie Mabrouki ◽

Katja Grabau ◽

Ralf Frederik Claas ◽

Agnes Rudowski ◽

...

Keyword(s):

Disease Type ◽

Cholesterol Homeostasis ◽

Niemann Pick Disease ◽

Sphingosine 1 Phosphate ◽

Type C ◽

Niemann Pick ◽

Pick Disease

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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

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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

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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

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Faculty Opinions recommendation of The adult form of Niemann-Pick disease type C.

Faculty Opinions – Post-Publication Peer Review of the Biomedical Literature ◽

10.3410/f.1085930.538703 ◽

2007 ◽

Author(s):

Peter Brown

Keyword(s):

Disease Type ◽

Adult Form ◽

Niemann Pick Disease ◽

Type C ◽

Niemann Pick ◽

Pick Disease

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Faculty Opinions recommendation of Liver and skin histopathology in adults with acid sphingomyelinase deficiency (Niemann-Pick disease type B).

Faculty Opinions – Post-Publication Peer Review of the Biomedical Literature ◽

10.3410/f.717597820.793453174 ◽

2012 ◽

Author(s):

Guadalupe Garcia-Tsao

Keyword(s):

Disease Type ◽

Acid Sphingomyelinase ◽

Type B ◽

Sphingomyelinase Deficiency ◽

Niemann Pick Disease ◽

Niemann Pick ◽

Pick Disease

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Faculty Opinions recommendation of Laboratory diagnosis of Niemann-Pick disease type C: the filipin staining test.

Faculty Opinions – Post-Publication Peer Review of the Biomedical Literature ◽

10.3410/f.725350592.793542531 ◽

2018 ◽

Author(s):

Barbara Borroni

Keyword(s):

Laboratory Diagnosis ◽

Disease Type ◽

Niemann Pick Disease ◽

Type C ◽

Niemann Pick ◽

Pick Disease

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Faculty Opinions recommendation of Diagnostic tests for Niemann-Pick disease type C (NP-C): A critical review.

Faculty Opinions – Post-Publication Peer Review of the Biomedical Literature ◽

10.3410/f.726446034.793542532 ◽

2018 ◽

Author(s):

Barbara Borroni

Keyword(s):

Critical Review ◽

Diagnostic Tests ◽

Disease Type ◽

Niemann Pick Disease ◽

Type C ◽

Niemann Pick ◽

Pick Disease

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Faculty Opinions recommendation of Cognition and anatomy of adult Niemann-Pick disease type C: Insights for the Alzheimer field.

Faculty Opinions – Post-Publication Peer Review of the Biomedical Literature ◽

10.3410/f.729491176.793542536 ◽

2018 ◽

Author(s):

Barbara Borroni

Keyword(s):

Disease Type ◽

Niemann Pick Disease ◽

Type C ◽

Niemann Pick ◽

Pick Disease

Download Full-text