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A therapy with miglustat, 2-hydroxypropyl-ß-cyclodextrin and allopregnanolone restores splenic cholesterol homeostasis in Niemann-pick disease type C1.
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.
Identification of Brain-Specific Treatment Effects in NPC1 Disease by Focusing on Cellular and Molecular Changes of Sphingosine-1-Phosphate Metabolism.
Gläser A, Hammerl F, Gräler MH, Coldewey SM, Völkner C, Frech MJ, Yang F, Luo J, Tönnies E, von Bohlen Und Halbach O, Brandt N, Heimes D, Neßlauer AM, Korenke GC, Owczarek-Lipska M, Neidhardt J, Rolfs A, Wree A, Witt M, Bräuer AU.
Gläser A, et al. Among authors: nesslauer am.
Int J Mol Sci. 2020 Jun 24;21(12):4502. doi: 10.3390/ijms21124502.
Int J Mol Sci. 2020.
PMID: 32599915
Free PMC article.
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Glucosylsphingosine Causes Hematological and Visceral Changes in Mice-Evidence for a Pathophysiological Role in Gaucher Disease.
Lukas J, Cozma C, Yang F, Kramp G, Meyer A, Neßlauer AM, Eichler S, Böttcher T, Witt M, Bräuer AU, Kropp P, Rolfs A.
Lukas J, et al. Among authors: nesslauer am.
Int J Mol Sci. 2017 Oct 20;18(10):2192. doi: 10.3390/ijms18102192.
Int J Mol Sci. 2017.
PMID: 29053611
Free PMC article.
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