Update on phase I study to evaluate the single- and multiple-dose pharmacokinetics of intravenous hydroxypropyl betacyclodextrin (HPβCD) in patients with Niemann-Pick disease type C (NPC-1) and the effects of dosing upon biomarkers of NPC disease

2021 ◽  
Vol 132 (2) ◽  
pp. S48
Author(s):  
Caroline Aimee Hastings ◽  
Benny Liu ◽  
Alan Boyd ◽  
Sharon Hrynkow
Keyword(s):  
Phase I ◽  
Phase I Study ◽  
Multiple Dose ◽  
Disease Type ◽  
Niemann Pick Disease ◽  
Type C ◽  
Niemann Pick ◽  
Multiple Dose Pharmacokinetics ◽  
Pick Disease

ANALYSIS OF CHOLESTEROL AND OXYSTEROL LEVELS IN NIEMANN PICK DISEASE TYPE C MICE AND PATIENTS

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

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

Nature ◽  
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 ◽  
Pick Disease
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