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

2019 ◽  
Author(s):  
Keyword(s):  
Disease Type ◽  
Orphan Designation ◽  
Beta Cyclodextrin ◽  
Niemann Pick Disease ◽  
Type C ◽  
Niemann Pick ◽  
Pick Disease

A novel HPLC–LS method to analyze Hydroxypropyl-beta-Cyclodextrin in urine. Application to child with Niemann–Pick disease, type C

2016 ◽  
Vol 33 (3) ◽  
pp. 417-418
Author(s):  
A. Matencio ◽  
M.A. Alcaraz-Gómez ◽  
C. Garcia-Hernandez-Gil ◽  
F. García-Carmona ◽  
B. Arias ◽  
...  
Keyword(s):  
Disease Type ◽  
Beta Cyclodextrin ◽  
Niemann Pick Disease ◽  
Type C ◽  
Niemann Pick ◽  
Hydroxypropyl Beta Cyclodextrin ◽  
Pick Disease

scholarly journals Effect of Maltosyl-Beta-cyclodextrin on in vitro and in vivo models of Niemann-Pick disease type C

2018 ◽  
Vol WCP2018 (0) ◽  
pp. PO3-8-13
Author(s):  
Nushrat Yasmin ◽  
Yoichi Ishitsuka ◽  
Yusei Yamada ◽  
Madoka Fukaura ◽  
Shuichi Nakahara ◽  
...  
Keyword(s):  
Disease Type ◽  
In Vivo Models ◽  
Beta Cyclodextrin ◽  
Niemann Pick Disease ◽  
Type C ◽  
Niemann Pick ◽  
Pick Disease

Intrathecal 2-Hydroxypropyl-Beta-Cyclodextrin (HPBCD) therapy in adult-onset Niemann-Pick Disease Type C (NPC)

2015 ◽  
Vol 357 ◽  
pp. e209 ◽  
Author(s):  
Y. Sakiyama ◽  
S. Shibata ◽  
H. Sanayama ◽  
S. Ono ◽  
M. Maekawa ◽  
...  
Keyword(s):  
Disease Type ◽  
Adult Onset ◽  
Beta Cyclodextrin ◽  
Niemann Pick Disease ◽  
Type C ◽  
Niemann Pick ◽  
Hydroxypropyl Beta Cyclodextrin ◽  
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
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