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

Niemann-Pick type C1 (NPC1) disease is a fatal neurovisceral disease for which there are no FDA approved treatments, though cyclodextrin (HPβCD) slows disease progression in preclinical models and in an early phase clinical trial. Our goal was to evaluate the mechanism of action of a previously desc...

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Pubblicato in:Biochim Biophys Acta Mol Cell Biol Lipids
Autori principali: Davidson, Jessica, Molitor, Elizabeth, Moores, Samantha, Gale, Sarah E., Subramanian, Kanagaraj, Jiang, Xuntian, Sidhu, Rohini, Kell, Pamela, Zhang, Jesse, Fujiwara, Hideji, Davidson, Cristin, Helquist, Paul, Melancon, Bruce J., Grigalunas, Michael, Liu, Gang, Salahi, Farbod, Wiest, Olaf, Xu, Xin, Porter, Forbes D., Pipalia, Nina H., Cruz, Dana L., Holson, Edward B., Schaffer, Jean E., Walkley, Steven U., Maxfield, Frederick R., Ory, Daniel S.
Natura: Artigo
Lingua:Inglês
Pubblicazione: 2019
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Article
Accesso online:https://ncbi.nlm.nih.gov/pmc/articles/PMC6679735/
https://ncbi.nlm.nih.gov/pubmed/31051283
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.bbalip.2019.04.011
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