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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. Neßlauer AM, et al. Among authors: brauer au. Lipids Health Dis. 2019 Jun 28;18(1):146. doi: 10.1186/s12944-019-1088-2. Lipids Health Dis. 2019. PMID: 31248418 Free PMC article.
Proteostasis regulators modulate proteasomal activity and gene expression to attenuate multiple phenotypes in Fabry disease.
Seemann S, Ernst M, Cimmaruta C, Struckmann S, Cozma C, Koczan D, Knospe AM, Haake LR, Citro V, Bräuer AU, Andreotti G, Cubellis MV, Fuellen G, Hermann A, Giese AK, Rolfs A, Lukas J. Seemann S, et al. Among authors: brauer au. Biochem J. 2020 Jan 31;477(2):359-380. doi: 10.1042/BCJ20190513. Biochem J. 2020. PMID: 31899485 Free PMC article.
73 results