Reduction of glutamate neurotoxicity: A novel therapeutic approach for Niemann-Pick disease, type C1

Molecular Genetics and Metabolism ◽

10.1016/j.ymgme.2021.11.008 ◽

2021 ◽

Author(s):

Antony Cougnoux ◽

Julia C. Yerger ◽

Mason Fellmeth ◽

Jenny Serra-Vinardell ◽

Fatemeh Navid ◽

...

Keyword(s):

Therapeutic Approach ◽

Disease Type ◽

Glutamate Neurotoxicity ◽

Niemann Pick Disease ◽

Niemann Pick ◽

Pick Disease ◽

Novel Therapeutic

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 ◽

Type C ◽

Niemann Pick ◽

Download Full-text

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 ◽

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 ◽

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 ◽

Download Full-text

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 ◽

Download Full-text

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 ◽

Download Full-text

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 ◽

Download Full-text

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 ◽

Download Full-text

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 ◽

Download Full-text

Update on safety and efficacy results for phase I/II trial of hydroxypropyl betacyclodextrin (HPâCD) administered intravenously in patients with Niemann-Pick disease type C1

Molecular Genetics and Metabolism ◽

10.1016/j.ymgme.2020.12.215 ◽

2021 ◽

Vol 132 (2) ◽

pp. S90

Author(s):

Julian A.J. Raiman ◽

Reena Sharma ◽

Martin Paucar ◽

Orna Staretz-Chacham ◽

Ronen Spiegel ◽

...

Keyword(s):

Phase I ◽

Disease Type ◽

Safety And Efficacy ◽

Niemann Pick Disease ◽

Niemann Pick ◽

Download Full-text