217 related articles for article (PubMed ID: 27307499)
21. Chronic cyclodextrin treatment of murine Niemann-Pick C disease ameliorates neuronal cholesterol and glycosphingolipid storage and disease progression.
Davidson CD; Ali NF; Micsenyi MC; Stephney G; Renault S; Dobrenis K; Ory DS; Vanier MT; Walkley SU
PLoS One; 2009 Sep; 4(9):e6951. PubMed ID: 19750228
[TBL] [Abstract][Full Text] [Related]
22. Long-term administration of intravenous Trappsol® Cyclo™ (HP-β-CD) results in clinical benefits and stabilization or slowing of disease progression in patients with Niemann-Pick disease type C1: Results of an international 48-week Phase I/II trial.
Sharma R; Hastings C; Staretz-Chacham O; Raiman J; Paucar M; Spiegel R; Murray B; Hurst B; Liu B; Kjems L; Hrynkow S
Mol Genet Metab Rep; 2023 Sep; 36():100988. PubMed ID: 37670901
[TBL] [Abstract][Full Text] [Related]
23. LC3 Immunostaining in the Inferior Olivary Nuclei of Cats With Niemann-Pick Disease Type C1 Is Associated With Patterned Purkinje Cell Loss.
Gurda BL; Bagel JH; Fisher SJ; Schultz ML; Lieberman AP; Hand P; Vite CH; Swain GP
J Neuropathol Exp Neurol; 2018 Mar; 77(3):229-245. PubMed ID: 29346563
[TBL] [Abstract][Full Text] [Related]
24. Long-Term Neuropsychological Outcomes from an Open-Label Phase I/IIa Trial of 2-Hydroxypropyl-β-Cyclodextrins (VTS-270) in Niemann-Pick Disease, Type C1.
Farmer CA; Thurm A; Farhat N; Bianconi S; Keener LA; Porter FD
CNS Drugs; 2019 Jul; 33(7):677-683. PubMed ID: 31187454
[TBL] [Abstract][Full Text] [Related]
25. 2-Hydroxypropyl-β-cyclodextrin is the active component in a triple combination formulation for treatment of Niemann-Pick C1 disease.
Davidson J; Molitor E; Moores S; Gale SE; Subramanian K; Jiang X; Sidhu R; Kell P; Zhang J; Fujiwara H; Davidson C; Helquist P; Melancon BJ; Grigalunas M; Liu G; Salahi F; Wiest O; Xu X; Porter FD; Pipalia NH; Cruz DL; Holson EB; Schaffer JE; Walkley SU; Maxfield FR; Ory DS
Biochim Biophys Acta Mol Cell Biol Lipids; 2019 Oct; 1864(10):1545-1561. PubMed ID: 31051283
[TBL] [Abstract][Full Text] [Related]
26. In Niemann-Pick C1 mouse models, glial-only expression of the normal gene extends survival much further than do changes in genetic background or treatment with hydroxypropyl-beta-cyclodextrin.
Marshall CA; Watkins-Chow DE; Palladino G; Deutsch G; Chandran K; Pavan WJ; Erickson RP
Gene; 2018 Feb; 643():117-123. PubMed ID: 29223359
[TBL] [Abstract][Full Text] [Related]
27. Transcriptome of HPβCD-treated Niemann-Pick disease type C1 cells highlights GPNMB as a biomarker for therapeutics.
Rodriguez-Gil JL; Baxter LL; Watkins-Chow DE; Johnson NL; Davidson CD; Carlson SR; Incao AA; ; Wallom KL; Farhat NY; Platt FM; Dale RK; Porter FD; Pavan WJ
Hum Mol Genet; 2021 Nov; 30(24):2456-2468. PubMed ID: 34296265
[TBL] [Abstract][Full Text] [Related]
28. In vitro evaluation of 2-hydroxyalkylated β-cyclodextrins as potential therapeutic agents for Niemann-Pick Type C disease.
Kondo Y; Tokumaru H; Ishitsuka Y; Matsumoto T; Taguchi M; Motoyama K; Higashi T; Arima H; Matsuo M; Higaki K; Ohno K; Irie T
Mol Genet Metab; 2016 Jul; 118(3):214-219. PubMed ID: 27184436
[TBL] [Abstract][Full Text] [Related]
29. Systemic administration of 2-hydroxypropyl-β-cyclodextrin to symptomatic Npc1-deficient mice slows cholesterol sequestration in the major organs and improves liver function.
Lopez AM; Terpack SJ; Posey KS; Liu B; Ramirez CM; Turley SD
Clin Exp Pharmacol Physiol; 2014 Oct; 41(10):780-7. PubMed ID: 25115571
[TBL] [Abstract][Full Text] [Related]
30. Cholesterol homeostatic responses provide biomarkers for monitoring treatment for the neurodegenerative disease Niemann-Pick C1 (NPC1).
Tortelli B; Fujiwara H; Bagel JH; Zhang J; Sidhu R; Jiang X; Yanjanin NM; Shankar RK; Carillo-Carasco N; Heiss J; Ottinger E; Porter FD; Schaffer JE; Vite CH; Ory DS
Hum Mol Genet; 2014 Nov; 23(22):6022-33. PubMed ID: 24964810
[TBL] [Abstract][Full Text] [Related]
31. In Vitro and In Vivo Evaluation of 6-O-α-Maltosyl-β-Cyclodextrin as a Potential Therapeutic Agent Against Niemann-Pick Disease Type C.
Yasmin N; Ishitsuka Y; Fukaura M; Yamada Y; Nakahara S; Ishii A; Kondo Y; Takeo T; Nakagata N; Motoyama K; Higashi T; Okada Y; Nishikawa J; Ichikawa A; Iohara D; Hirayama F; Higaki K; Ohno K; Matsuo M; Irie T
Int J Mol Sci; 2019 Mar; 20(5):. PubMed ID: 30845767
[TBL] [Abstract][Full Text] [Related]
32. Normalization of cholesterol homeostasis by 2-hydroxypropyl-β-cyclodextrin in neurons and glia from Niemann-Pick C1 (NPC1)-deficient mice.
Peake KB; Vance JE
J Biol Chem; 2012 Mar; 287(12):9290-8. PubMed ID: 22277650
[TBL] [Abstract][Full Text] [Related]
33. A differential proteomics study of cerebrospinal fluid from individuals with Niemann-Pick disease, Type C1.
Li W; Pergande MR; Crutchfield CA; Searle BC; Backlund PS; Picache JA; Burkert K; Yanjanin-Farhat NM; Blank PS; Toth CL; Wassif CA; Porter FD; Cologna SM
Proteomics; 2023 Jun; 23(11):e2200378. PubMed ID: 36638187
[TBL] [Abstract][Full Text] [Related]
34. Defective Cytochrome P450-Catalysed Drug Metabolism in Niemann-Pick Type C Disease.
Nicoli ER; Al Eisa N; Cluzeau CV; Wassif CA; Gray J; Burkert KR; Smith DA; Morris L; Cologna SM; Peer CJ; Sissung TM; Uscatu CD; Figg WD; Pavan WJ; Vite CH; Porter FD; Platt FM
PLoS One; 2016; 11(3):e0152007. PubMed ID: 27019000
[TBL] [Abstract][Full Text] [Related]
35. Increased Regenerative Capacity of the Olfactory Epithelium in Niemann-Pick Disease Type C1.
Meyer A; Wree A; Günther R; Holzmann C; Schmitt O; Rolfs A; Witt M
Int J Mol Sci; 2017 Apr; 18(4):. PubMed ID: 28383485
[TBL] [Abstract][Full Text] [Related]
36. 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]
37. Visual evoked potentials of Niemann-Pick type C1 mice reveal an impairment of the visual pathway that is rescued by 2-hydroxypropyl-ß-cyclodextrin.
Palladino G; Loizzo S; Fortuna A; Canterini S; Palombi F; Erickson RP; Mangia F; Fiorenza MT
Orphanet J Rare Dis; 2015 Oct; 10():133. PubMed ID: 26458950
[TBL] [Abstract][Full Text] [Related]
38. Application of a glycinated bile acid biomarker for diagnosis and assessment of response to treatment in Niemann-pick disease type C1.
Sidhu R; Kell P; Dietzen DJ; Farhat NY; Do AND; Porter FD; Berry-Kravis E; Reunert J; Marquardt T; Giugliani R; Lourenço CM; Wang RY; Movsesyan N; Plummer E; Schaffer JE; Ory DS; Jiang X
Mol Genet Metab; 2020 Dec; 131(4):405-417. PubMed ID: 33257258
[TBL] [Abstract][Full Text] [Related]
39. The susceptibility of cochlear outer hair cells to cyclodextrin is not related to their electromotile activity.
Zhou Y; Takahashi S; Homma K; Duan C; Zheng J; Cheatham MA; Zheng J
Acta Neuropathol Commun; 2018 Sep; 6(1):98. PubMed ID: 30249300
[TBL] [Abstract][Full Text] [Related]
40. Low Risk Profile of Long-Term Repeated Lumbar Puncture for Intrathecal Delivery of 2-Hydroxypropyl-Beta-Cyclodextrin in Patients With Niemann-Pick Type C.
Albert OK; Friedmann K; Jaeger R; Berry-Kravis E
Pediatr Neurol; 2023 Jul; 144():99-103. PubMed ID: 37201244
[TBL] [Abstract][Full Text] [Related]
[Previous] [Next] [New Search]