Efficacy and ototoxicity of different cyclodextrins in Niemann–Pick C disease
Annals of Clinical and Translational Neurology2016Vol. 3(5), pp. 366–380
Citations Over TimeTop 10% of 2016 papers
Cristin Davidson, Yonatan I. Fishman, István Puskás, Julianna Szemán, Tamás Sohajda, Leslie A. McCauliff, Jakub Sikora, Judith Storch, Marie T. Vanier, Lajos Szente, Steven U. Walkley, Kostantin Dobrenis
Abstract
CDs other than HPβCD identified here may provide disease amelioration without ototoxicity and merit long-term treatment studies. While direct interactions of CD-UC are thought central to the mechanism of correction, the data show that this does not strictly correlate with complexation ability and suggest interactions with other NPC disease-relevant substrates should be considered.
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