scholarly journals Use of 2-Hydroxypropyl-Beta-Cyclodextrin for Niemann-Pick Type C Disease

2018 ◽  
Author(s):  
Juan Eduardo Megías-Vericat ◽  
María José Company-Albir ◽  
Ana Alejandra García-Robles ◽  
José Luis Poveda
Keyword(s):  
Beta Cyclodextrin ◽  
Type C ◽  
Niemann Pick ◽  
Hydroxypropyl Beta Cyclodextrin

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

Early experience with compassionate use of 2 hydroxypropyl-beta-cyclodextrin for Niemann–Pick type C disease: review of initial published cases

2017 ◽  
Vol 38 (5) ◽  
pp. 727-743 ◽  
Author(s):  
Juan Eduardo Megías-Vericat ◽  
Ana García-Robles ◽  
María José Company-Albir ◽  
María José Fernández-Megía ◽  
Francisco Carlos Pérez-Miralles ◽  
...  
Keyword(s):  
Early Experience ◽  
Compassionate Use ◽  
Beta Cyclodextrin ◽  
Type C ◽  
Niemann Pick ◽  
Hydroxypropyl Beta Cyclodextrin

Use of 2 hydroxypropyl-beta-cyclodextrin therapy in two adult Niemann Pick Type C patients

2016 ◽  
Vol 366 ◽  
pp. 65-67 ◽  
Author(s):  
Ana Alejandra García-Robles ◽  
María José Company-Albir ◽  
Juan Eduardo Megías-Vericat ◽  
María José Fernández-Megía ◽  
Francisco Carlos Pérez-Miralles ◽  
...  
Keyword(s):  
Beta Cyclodextrin ◽  
Type C ◽  
Niemann Pick ◽  
Hydroxypropyl Beta Cyclodextrin

scholarly journals Intrathecal 2-hydroxypropyl-beta-cyclodextrin in a single patient with Niemann–Pick C1

2015 ◽  
Vol 116 (1-2) ◽  
pp. 75-79 ◽  
Author(s):  
Timothy J. Maarup ◽  
Agnes H. Chen ◽  
Forbes D. Porter ◽  
Nicole Y. Farhat ◽  
Daniel S. Ory ◽  
...  
Keyword(s):  
Single Patient ◽  
Beta Cyclodextrin ◽  
Niemann Pick ◽  
Hydroxypropyl Beta Cyclodextrin

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
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