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...

Descripció completa

Guardat en:
Dades bibliogràfiques
Publicat a:Biochim Biophys Acta Mol Cell Biol Lipids
Autors principals: 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.
Format: Artigo
Idioma:Inglês
Publicat: 2019
Matèries:
Article
Accés en línia: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
Etiquetes: Afegir etiqueta
Sense etiquetes, Sigues el primer a etiquetar aquest registre!

Ítems similars