(Q38627151)
Statements
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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 (English)
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Christopher P Austin
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Daniel S Ory
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Elizabeth A Ottinger
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Nicole Yanjanin Farhat
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Kelly A King
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Xuntian Jiang
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Lisa Weissfeld
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Elizabeth Berry-Kravis
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Simona Bianconi
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Lee Ann Keener
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Ravichandran Rao
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Ariane Soldatos
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Kimberly A Walters
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Xin Xu
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Audrey Thurm
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Beth Solomon
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Bernardus N Machielse
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Mark Kao
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Steven A Silber
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John C McKew
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Carmen C Brewer
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Charles H Vite
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Steven U Walkley
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Forbes D Porter
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10 August 2017
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Identifiers
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