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Intrathecal 2-hydroxypropyl-β-cyclodextrin decreases neurological disease progression in Niemann-Pick disease, type C1: a non-randomised, open-label, phase 1–2 trial
Author(s):
Daniel S. Ory
,
Elizabeth Ottinger
,
Nicole Farhat
,
Kelly A. King
,
Xuntian Jiang
,
Lisa Weissfeld
,
Elizabeth Berry-Kravis
,
Cristin Davidson
,
Simona Bianconi
,
Lee Keener
,
Ravichandran Rao
,
Ariane Soldatos
,
Rohini Sidhu
,
Kimberly A Walters
,
Xin Xu
,
Audrey Thurm
,
Beth Solomon
,
William J. Pavan
,
Bernardus N. Machielse Drs
,
Mark L Kao
,
Steven Silber
,
John C McKew
,
Carmen Brewer
,
Charles Vite
,
Steven U Walkley
,
Christopher Austin
,
Forbes D Porter
Publication date:
2017-10-01
Journal:
The Lancet
Publisher:
Elsevier BV
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Author and article information
Journal
DOI::
10.1016/S0140-6736(17)31465-4
License:
https://www.elsevier.com/tdm/userlicense/1.0/
Data availability:
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