2-Hydroxypropyl-β-cyclodextrin is the active component in a triple combination formulation for treatment of Niemann-Pick C1 disease

Jessica Davidson, Elizabeth Molitor, Samantha Moores, Sarah E. Gale, Kanagaraj Subramanian, Xuntian Jiang, Rohini Sidhu, Pamela Kell, J. Zhang, Hideji Fujiwara, Cristin Davidson, Paul Helquist, Bruce J. Melancon, Michael Grigalunas, Gang Liu, Farbod Salahi, Olaf Wiest, Xin Xu, Forbes D. Porter, Nina H. PipaliaDana L. Cruz, Edward B. Holson, Jean E. Schaffer, Steven U. Walkley, Frederick R. Maxfield, Daniel S. Ory

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Medicine & Life Sciences