Pharmacology & ToxicologybioRxivPreprint
Elucidating the mechanism of cyclodextrins in the treatment of Niemann-Pick Disease Type C using crosslinked 2-hydroxypropyl-β-cyclodextrin
D. Carradori, H. Chen, B. Werner…·J.-C. Leroux
This study investigates the mechanism of crosslinked 2-hydroxypropyl-β-cyclodextrin (HPβCD) in treating Niemann-Pick Disease Type C and evaluates its in vivo efficacy with MRIg-FUS to enhance brain penetration.
DOI 10.1101/2020.07.31.230136