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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
Authors
Daniel Ory,Elizabeth Ottinger
Nicole Farhat,Kelly King,Xuntian Jiang,Lisa Weissfeld,Elizabeth Berry‐Kravis,Cristin Davidson,Simona Bianconi,Lee Keener,Ravichandran Rao,Ariane Soldatos,Rohini Sidhu,Kimberly Walters,Xin Xu,Audrey Thurm,Beth Solomon,William Pavan,Bernardus Machielse,Mark Kao,Steven Silber,John McKew,Carmen Brewer,Charles Vite,Steven Walkley,Christopher Austin
+24 authors
,Forbes PorterJournal
Published
Aug 11, 2017
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