Intrathecal 2-hydroxypropyl-β-cyclodextrin decreases neurological disease progression in Niemann-Pick disease, type C1: a non-randomised, open-label, phase 1–2 trial
Daniel S. Ory, Elizabeth A. Ottinger, Nicole Yanjanin Farhat, Kelly A. King, Xuntian Jiang, Lisa Weissfeld, Elizabeth Berry-Kravis, Cristin D. 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 KaoSteven A. Silber, John C. McKew, Carmen C. Brewer, Charles H. Vite, Steven U. Walkley, Christopher P. Austin, Forbes D. Porter
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