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 S. Ory, Elizabeth A. Ottinger, Nicole Y. Farhat, Kelly King, Xuntian Jiang, Lisa A. Weissfeld, Elizabeth Berry‐Kravis, Cristin Davidson, Simona Bianconi, Lee Ann Keener, Ravichandran Rao, Ariane Soldatos, Rohini Sidhu, Kimberly A. Walters, Xin Xu, Audrey Thurm, Beth Solomon, William J. Pavan, Bernardus N Machielse, Mark Kao, Steven A. Silber, John C. McKew, Carmen C. Brewer, Charles H. Vite, Steven U. Walkley, Christopher P. Austin, Forbes D. Porter - The Lancet 2017 cited by 325