These tools will no longer be maintained as of December 31, 2024. Archived website can be found here. PubMed4Hh GitHub repository can be found here. Contact NLM Customer Service if you have questions.
181 related articles for article (PubMed ID: 28414792)
1. Characterization of hydroxypropyl-beta-cyclodextrins used in the treatment of Niemann-Pick Disease type C1. Yergey AL; Blank PS; Cologna SM; Backlund PS; Porter FD; Darling AJ PLoS One; 2017; 12(4):e0175478. PubMed ID: 28414792 [TBL] [Abstract][Full Text] [Related]
2. 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]
3. 2-Hydroxypropyl-β-cyclodextrins and the Blood-Brain Barrier: Considerations for Niemann-Pick Disease Type C1. Calias P Curr Pharm Des; 2017; 23(40):6231-6238. PubMed ID: 29065825 [TBL] [Abstract][Full Text] [Related]
4. 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]
6. Hydroxypropyl-beta and -gamma cyclodextrins rescue cholesterol accumulation in Niemann-Pick C1 mutant cell via lysosome-associated membrane protein 1. Singhal A; Szente L; Hildreth JEK; Song B Cell Death Dis; 2018 Oct; 9(10):1019. PubMed ID: 30282967 [TBL] [Abstract][Full Text] [Related]
7. 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]
8. 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]
9. 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]
10. Methyl-β-cyclodextrin restores impaired autophagy flux in Niemann-Pick C1-deficient cells through activation of AMPK. Dai S; Dulcey AE; Hu X; Wassif CA; Porter FD; Austin CP; Ory DS; Marugan J; Zheng W Autophagy; 2017 Aug; 13(8):1435-1451. PubMed ID: 28613987 [TBL] [Abstract][Full Text] [Related]
11. Intrathecal 2-hydroxypropyl-beta-cyclodextrin in a single patient with Niemann-Pick C1. Maarup TJ; Chen AH; Porter FD; Farhat NY; Ory DS; Sidhu R; Jiang X; Dickson PI Mol Genet Metab; 2015; 116(1-2):75-9. PubMed ID: 26189084 [TBL] [Abstract][Full Text] [Related]
12. 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]
13. 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]
14. 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]
15. Investigating the Mechanism of Cyclodextrins in the Treatment of Niemann-Pick Disease Type C Using Crosslinked 2-Hydroxypropyl-β-cyclodextrin. Carradori D; Chen H; Werner B; Shah AS; Leonardi C; Usuelli M; Mezzenga R; Platt F; Leroux JC Small; 2020 Nov; 16(46):e2004735. PubMed ID: 33079457 [TBL] [Abstract][Full Text] [Related]
16. 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]
17. Modeling Niemann-Pick disease type C1 in zebrafish: a robust platform for Tseng WC; Loeb HE; Pei W; Tsai-Morris CH; Xu L; Cluzeau CV; Wassif CA; Feldman B; Burgess SM; Pavan WJ; Porter FD Dis Model Mech; 2018 Aug; 11(9):. PubMed ID: 30135069 [TBL] [Abstract][Full Text] [Related]
18. 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]
19. Accumulation of alkyl-lysophosphatidylcholines in Niemann-Pick disease type C1. Mishra S; Kell P; Scherrer D; Dietzen DJ; Vite CH; Berry-Kravis E; Davidson C; Cologna SM; Porter FD; Ory DS; Jiang X J Lipid Res; 2024 Aug; 65(8):100600. PubMed ID: 39048052 [TBL] [Abstract][Full Text] [Related]
20. Expanded access with intravenous hydroxypropyl-β-cyclodextrin to treat children and young adults with Niemann-Pick disease type C1: a case report analysis. Hastings C; Vieira C; Liu B; Bascon C; Gao C; Wang RY; Casey A; Hrynkow S Orphanet J Rare Dis; 2019 Oct; 14(1):228. PubMed ID: 31639011 [TBL] [Abstract][Full Text] [Related] [Next] [New Search]