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 ◽

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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 ◽

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Orphan designation: Hydroxy-propyl-beta-cyclodextrin, Treatment of Niemann-Pick disease, type C

Case Medical Research ◽

10.31525/cmr-da7a89 ◽

2019 ◽

Author(s):

Keyword(s):

Disease Type ◽

Orphan Designation ◽

Beta Cyclodextrin ◽

Niemann Pick Disease ◽

Type C ◽

Niemann Pick ◽

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Orphan designation: miglustat, Treatment of Niemann-Pick disease, type C

Case Medical Research ◽

10.31525/cmr-bc6b4a ◽

2019 ◽

Author(s):

Keyword(s):

Disease Type ◽

Orphan Designation ◽

Niemann Pick Disease ◽

Type C ◽

Niemann Pick ◽

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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 ◽

Type C ◽

Niemann Pick ◽

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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 ◽

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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 ◽

Niemann Pick Disease ◽

Type C ◽

Niemann Pick ◽

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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 ◽

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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 ◽

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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 ◽

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Tonic prime-boost of STING signalling mediates Niemann–Pick disease type C

Nature ◽

10.1038/s41586-021-03762-2 ◽

2021 ◽

Author(s):

Ting-Ting Chu ◽

Xintao Tu ◽

Kun Yang ◽

Jianjun Wu ◽

Joyce J. Repa ◽

...

Keyword(s):

Disease Type ◽

Niemann Pick Disease ◽

Type C ◽

Niemann Pick ◽

Download Full-text