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6. 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]
7. Differential mode of cholesterol inclusion with 2-hydroxypropyl-cyclodextrins increases safety margin in treatment of Niemann-Pick disease type C. Yamada Y; Ishitsuka Y; Kondo Y; Nakahara S; Nishiyama A; Takeo T; Nakagata N; Motoyama K; Higashi T; Arima H; Kamei S; Shuto T; Kai H; Hayashino Y; Sugita M; Kikuchi T; Hirata F; Miwa T; Takeda H; Orita Y; Seki T; Ohta T; Kurauchi Y; Katsuki H; Matsuo M; Higaki K; Ohno K; Matsumoto S; Era T; Irie T Br J Pharmacol; 2021 Jul; 178(13):2727-2746. PubMed ID: 33782944 [TBL] [Abstract][Full Text] [Related]
8. 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]
9. Intracerebroventricular Treatment with 2-Hydroxypropyl-β-Cyclodextrin Decreased Cerebellar and Hepatic Glycoprotein Nonmetastatic Melanoma Protein B (GPNMB) Expression in Niemann-Pick Disease Type C Model Mice. Fukaura M; Ishitsuka Y; Shirakawa S; Ushihama N; Yamada Y; Kondo Y; Takeo T; Nakagata N; Motoyama K; Higashi T; Arima H; Kurauchi Y; Seki T; Katsuki H; Higaki K; Matsuo M; Irie T Int J Mol Sci; 2021 Jan; 22(1):. PubMed ID: 33466390 [TBL] [Abstract][Full Text] [Related]
11. Collaborative development of 2-hydroxypropyl-β-cyclodextrin for the treatment of Niemann-Pick type C1 disease. Ottinger EA; Kao ML; Carrillo-Carrasco N; Yanjanin N; Shankar RK; Janssen M; Brewster M; Scott I; Xu X; Cradock J; Terse P; Dehdashti SJ; Marugan J; Zheng W; Portilla L; Hubbs A; Pavan WJ; Heiss J; Vite CH; Walkley SU; Ory DS; Silber SA; Porter FD; Austin CP; McKew JC Curr Top Med Chem; 2014; 14(3):330-9. PubMed ID: 24283970 [TBL] [Abstract][Full Text] [Related]
12. 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]
13. Long-Term Treatment of Niemann-Pick Type C1 Disease With Intrathecal 2-Hydroxypropyl-β-Cyclodextrin. Berry-Kravis E; Chin J; Hoffmann A; Winston A; Stoner R; LaGorio L; Friedmann K; Hernandez M; Ory DS; Porter FD; O'Keefe JA Pediatr Neurol; 2018 Mar; 80():24-34. PubMed ID: 29429782 [TBL] [Abstract][Full Text] [Related]
14. 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]
15. Pharmacokinetics and distribution of 2-hydroxypropyl-β-cyclodextrin following a single intrathecal dose to cats. Kao ML; Stellar S; Solon E; Lordi A; Kasica N; Swain G; Bagel JH; Gurda BL; Vite CH J Inherit Metab Dis; 2020 May; 43(3):618-634. PubMed ID: 31707730 [TBL] [Abstract][Full Text] [Related]
16. Use of 2 hydroxypropyl-beta-cyclodextrin therapy in two adult Niemann Pick Type C patients. García-Robles AA; Company-Albir MJ; Megías-Vericat JE; Fernández-Megía MJ; Pérez-Miralles FC; López-Briz E; Alcalá-Vicente C; Galeano I; Casanova B; Poveda JL J Neurol Sci; 2016 Jul; 366():65-67. PubMed ID: 27288778 [No Abstract] [Full Text] [Related]
17. Intracisternal cyclodextrin prevents cerebellar dysfunction and Purkinje cell death in feline Niemann-Pick type C1 disease. Vite CH; Bagel JH; Swain GP; Prociuk M; Sikora TU; Stein VM; O'Donnell P; Ruane T; Ward S; Crooks A; Li S; Mauldin E; Stellar S; De Meulder M; Kao ML; Ory DS; Davidson C; Vanier MT; Walkley SU Sci Transl Med; 2015 Feb; 7(276):276ra26. PubMed ID: 25717099 [TBL] [Abstract][Full Text] [Related]
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19. 2-hydroxypropyl-beta-cyclodextrin raises hearing threshold in normal cats and in cats with Niemann-Pick type C disease. Ward S; O'Donnell P; Fernandez S; Vite CH Pediatr Res; 2010 Jul; 68(1):52-6. PubMed ID: 20357695 [TBL] [Abstract][Full Text] [Related]
20. Anesthetic management of pediatric patients with Niemann-Pick disease type C for intrathecal 2-hydroxypropyl-β-cyclodextrin injection. Ulloa ML; Froyshteter AB; Kret LN; Chang DP; Sarah GE; McCarthy RJ; Barnes SD; Berry-Kravis EM Paediatr Anaesth; 2020 Jul; 30(7):766-772. PubMed ID: 32349180 [TBL] [Abstract][Full Text] [Related] [Next] [New Search]