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