A therapy with miglustat, 2-hydroxypropyl-ß-cyclodextrin and allopregnanolone restores splenic cholesterol homeostasis in Niemann-pick disease type C1. - Neßlauer AM; Gläser A; Gräler M; Engelmann R; Müller-Hilke B; Frank M; Burstein C; Rolfs A; Neidhardt J; Wree A; Witt M; Bräuer AU (Lipids in health and disease, 2019)