217 related articles for article (PubMed ID: 24343124)
1. Altered transition metal homeostasis in Niemann-Pick disease, type C1.
Hung YH; Faux NG; Killilea DW; Yanjanin N; Firnkes S; Volitakis I; Ganio G; Walterfang M; Hastings C; Porter FD; Ory DS; Bush AI
Metallomics; 2014 Mar; 6(3):542-53. PubMed ID: 24343124
[TBL] [Abstract][Full Text] [Related]
2. Improved neuroprotection using miglustat, curcumin and ibuprofen as a triple combination therapy in Niemann-Pick disease type C1 mice.
Williams IM; Wallom KL; Smith DA; Al Eisa N; Smith C; Platt FM
Neurobiol Dis; 2014 Jul; 67():9-17. PubMed ID: 24631719
[TBL] [Abstract][Full Text] [Related]
3. Human and mouse neuroinflammation markers in Niemann-Pick disease, type C1.
Cologna SM; Cluzeau CV; Yanjanin NM; Blank PS; Dail MK; Siebel S; Toth CL; Wassif CA; Lieberman AP; Porter FD
J Inherit Metab Dis; 2014 Jan; 37(1):83-92. PubMed ID: 23653225
[TBL] [Abstract][Full Text] [Related]
4. Identification of Niemann-Pick C1 disease biomarkers through sphingolipid profiling.
Fan M; Sidhu R; Fujiwara H; Tortelli B; Zhang J; Davidson C; Walkley SU; Bagel JH; Vite C; Yanjanin NM; Porter FD; Schaffer JE; Ory DS
J Lipid Res; 2013 Oct; 54(10):2800-14. PubMed ID: 23881911
[TBL] [Abstract][Full Text] [Related]
5. Evaluation of Two Liver Treatment Strategies in a Mouse Model of Niemann-Pick-Disease Type C1.
Ebner L; Gläser A; Bräuer A; Witt M; Wree A; Rolfs A; Frank M; Vollmar B; Kuhla A
Int J Mol Sci; 2018 Mar; 19(4):. PubMed ID: 29587349
[TBL] [Abstract][Full Text] [Related]
6. Reduced cerebellar neurodegeneration after combined therapy with cyclodextrin/allopregnanolone and miglustat in NPC1: a mouse model of Niemann-Pick type C1 disease.
Maass F; Petersen J; Hovakimyan M; Schmitt O; Witt M; Hawlitschka A; Lukas J; Rolfs A; Wree A
J Neurosci Res; 2015 Mar; 93(3):433-42. PubMed ID: 25400034
[TBL] [Abstract][Full Text] [Related]
7. 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
Int J Mol Sci; 2020 Jun; 21(12):. PubMed ID: 32599915
[TBL] [Abstract][Full Text] [Related]
8. Normalization of Hepatic Homeostasis in the
Munkacsi AB; Hammond N; Schneider RT; Senanayake DS; Higaki K; Lagutin K; Bloor SJ; Ory DS; Maue RA; Chen FW; Hernandez-Ono A; Dahlson N; Repa JJ; Ginsberg HN; Ioannou YA; Sturley SL
J Biol Chem; 2017 Mar; 292(11):4395-4410. PubMed ID: 28031458
[TBL] [Abstract][Full Text] [Related]
9. Differential Effects of 2-Hydroxypropyl-Cyclodextrins on Lipid Accumulation in
Hoque S; Kondo Y; Sakata N; Yamada Y; Fukaura M; Higashi T; Motoyama K; Arima H; Higaki K; Hayashi A; Komiya T; Ishitsuka Y; Irie T
Int J Mol Sci; 2020 Jan; 21(3):. PubMed ID: 32019132
[TBL] [Abstract][Full Text] [Related]
10. Reduction of glutamate neurotoxicity: A novel therapeutic approach for Niemann-Pick disease, type C1.
Cougnoux A; Yerger JC; Fellmeth M; Serra-Vinardell J; Navid F; Wassif CA; Cawley NX; Porter FD
Mol Genet Metab; 2021 Dec; 134(4):330-336. PubMed ID: 34802899
[TBL] [Abstract][Full Text] [Related]
11. Miglustat therapy in the French cohort of paediatric patients with Niemann-Pick disease type C.
Héron B; Valayannopoulos V; Baruteau J; Chabrol B; Ogier H; Latour P; Dobbelaere D; Eyer D; Labarthe F; Maurey H; Cuisset JM; de Villemeur TB; Sedel F; Vanier MT
Orphanet J Rare Dis; 2012 Jun; 7():36. PubMed ID: 22676771
[TBL] [Abstract][Full Text] [Related]
12. Niemann-Pick disease type C1 is a sphingosine storage disease that causes deregulation of lysosomal calcium.
Lloyd-Evans E; Morgan AJ; He X; Smith DA; Elliot-Smith E; Sillence DJ; Churchill GC; Schuchman EH; Galione A; Platt FM
Nat Med; 2008 Nov; 14(11):1247-55. PubMed ID: 18953351
[TBL] [Abstract][Full Text] [Related]
13. Localization of Niemann-Pick C1 protein in astrocytes: implications for neuronal degeneration in Niemann- Pick type C disease.
Patel SC; Suresh S; Kumar U; Hu CY; Cooney A; Blanchette-Mackie EJ; Neufeld EB; Patel RC; Brady RO; Patel YC; Pentchev PG; Ong WY
Proc Natl Acad Sci U S A; 1999 Feb; 96(4):1657-62. PubMed ID: 9990080
[TBL] [Abstract][Full Text] [Related]
14. Adult Niemann-Pick disease type C in France: clinical phenotypes and long-term miglustat treatment effect.
Nadjar Y; Hütter-Moncada AL; Latour P; Ayrignac X; Kaphan E; Tranchant C; Cintas P; Degardin A; Goizet C; Laurencin C; Martzolff L; Tilikete C; Anheim M; Audoin B; Deramecourt V; De Gaillarbois TD; Roze E; Lamari F; Vanier MT; Héron B
Orphanet J Rare Dis; 2018 Oct; 13(1):175. PubMed ID: 30285904
[TBL] [Abstract][Full Text] [Related]
15. 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 Health Dis; 2019 Jun; 18(1):146. PubMed ID: 31248418
[TBL] [Abstract][Full Text] [Related]
16. Intravenous 2-hydroxypropyl-β-cyclodextrin (Trappsol® Cyclo™) demonstrates biological activity and impacts cholesterol metabolism in the central nervous system and peripheral tissues in adult subjects with Niemann-Pick Disease Type C1: Results of a phase 1 trial.
Hastings C; Liu B; Hurst B; Cox GF; Hrynkow S
Mol Genet Metab; 2022 Dec; 137(4):309-319. PubMed ID: 36279795
[TBL] [Abstract][Full Text] [Related]
17. Different Trafficking Phenotypes of Niemann-Pick C1 Gene Mutations Correlate with Various Alterations in Lipid Storage, Membrane Composition and Miglustat Amenability.
Brogden G; Shammas H; Walters F; Maalouf K; Das AM; Naim HY; Rizk S
Int J Mol Sci; 2020 Mar; 21(6):. PubMed ID: 32204338
[TBL] [Abstract][Full Text] [Related]
18. Quantitative proteomic analysis of Niemann-Pick disease, type C1 cerebellum identifies protein biomarkers and provides pathological insight.
Cologna SM; Jiang XS; Backlund PS; Cluzeau CV; Dail MK; Yanjanin NM; Siebel S; Toth CL; Jun HS; Wassif CA; Yergey AL; Porter FD
PLoS One; 2012; 7(10):e47845. PubMed ID: 23144710
[TBL] [Abstract][Full Text] [Related]
19. Assessment of FDA-Approved Drugs as a Therapeutic Approach for Niemann-Pick Disease Type C1 Using Patient-Specific iPSC-Based Model Systems.
Völkner C; Pantoom S; Liedtke M; Lukas J; Hermann A; Frech MJ
Cells; 2022 Jan; 11(3):. PubMed ID: 35159129
[TBL] [Abstract][Full Text] [Related]
20. Neurological Dysfunction in Early Maturity of a Model for Niemann-Pick C1 Carrier Status.
Hung YH; Walterfang M; Churilov L; Bray L; Jacobson LH; Barnham KJ; Jones NC; O'Brien TJ; Velakoulis D; Bush AI
Neurotherapeutics; 2016 Jul; 13(3):614-22. PubMed ID: 26942423
[TBL] [Abstract][Full Text] [Related]
[Next] [New Search]
