Intrathecal 2-hydroxypropyl-β-cyclodextrin decreases neurological disease progression in Niemann-Pick disease, type C1: a non-randomised, open-label, phase 1–2 trial
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
Published in The Lancet (British edition) (14.10.2017)
Published in The Lancet (British edition) (14.10.2017)
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Journal Article
Intrathecal 2-hydroxypropyl-β-cyclodextrin decreases neurological disease progression in Niemann-Pick Disease, type C1: an ad-hoc analysis of a non-randomized, open-label, phase 1/2 trial
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 Drs, 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.
Published in The Lancet (British edition) (10.08.2017)
Published in The Lancet (British edition) (10.08.2017)
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Journal Article