NDLI: Long-Term Treatment of Niemann-Pick Type C1 Disease With Intrathecal 2-Hydroxypropyl-β-Cyclodextrin

Content Provider	Scilit
Author	O'Keefe, Joan A. Berry-Kravis, Elizabeth Chin, Jamie Hoffmann, Anne Winston, Amy Stoner, Robin LaGorio, Lisa Friedmann, Katherine Hernandez, Mariana Ory, Daniel S. Porter, Forbes D.
Copyright Year	2018
Description	Journal: Pediatric neurology These trajectories are not consistent with the typical trajectory of the disease and suggest that intrathecal 2-hydoxypropyl-β-cyclodextrin has stabilized the disease over an extended period of time, supporting the current phase 2/3 controlled registration trial with VTS-270.
Related Links	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5857219/pdf
Ending Page	34
Page Count	11
Starting Page	24
ISSN	08878994
e-ISSN	18735150
DOI	10.1016/j.pediatrneurol.2017.12.014
Journal	Pediatric neurology
Volume Number	80
Language	English
Publisher	Elsevier BV
Publisher Date	2018-03-01
Access Restriction	Open
Subject Keyword	Journal: Pediatric neurology Radiology, Nuclear Medicine and Imaging Niemann-pick Type C Lysosomal Storage Disorder Neurodegenerative Disease
Content Type	Text
Resource Type	Article
Subject	Neurology Neurology (clinical) Pediatrics, Perinatology and Child Health Developmental Neuroscience

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Long-Term Treatment of Niemann-Pick Type C1 Disease With Intrathecal 2-Hydroxypropyl-β-Cyclodextrin