Intrathecal 2-hydroxypropyl-β-cyclodextrin decreases neurological disease progression in Niemann-Pick disease, type C1: a non-randomised, open-label, phase 1

Intrathecal 2-hydroxypropyl-β-cyclodextrin decreases neurological disease progression in Niemann-Pick disease, type C1: a non-randomised, open-label, phase 1–2 trial

Published:2017-10 Issue:10104 Volume:390 Page:1758-1768
ISSN:0140-6736
Container-title:The Lancet
language:en
Short-container-title:The Lancet

Author:

Ory Daniel S,Ottinger Elizabeth A,Farhat Nicole Yanjanin,King Kelly A,Jiang Xuntian,Weissfeld Lisa,Berry-Kravis Elizabeth,Davidson Cristin D,Bianconi Simona,Keener Lee Ann,Rao Ravichandran,Soldatos Ariane,Sidhu Rohini,Walters Kimberly A,Xu Xin,Thurm Audrey,Solomon Beth,Pavan William J,Machielse Bernardus N,Kao Mark,Silber Steven A,McKew John C,Brewer Carmen C,Vite Charles H,Walkley Steven U,Austin Christopher P,Porter Forbes D

Publisher

Elsevier BV

Subject

General Medicine

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