BIOMARKERS

Molecular Biopsy of Human Tumors

- a resource for Precision Medicine *

283 related articles for article (PubMed ID: 31639011)

  • 1. 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]  

  • 2. Intrathecal 2-hydroxypropyl-β-cyclodextrin decreases neurological disease progression in Niemann-Pick disease, type C1: a non-randomised, open-label, phase 1-2 trial.
    Ory DS; Ottinger EA; Farhat NY; King KA; Jiang X; Weissfeld L; Berry-Kravis E; Davidson CD; Bianconi S; Keener LA; Rao R; Soldatos A; Sidhu R; Walters KA; Xu X; Thurm A; Solomon B; Pavan WJ; Machielse BN; Kao M; Silber SA; McKew JC; Brewer CC; Vite CH; Walkley SU; Austin CP; Porter FD
    Lancet; 2017 Oct; 390(10104):1758-1768. PubMed ID: 28803710
    [TBL] [Abstract][Full Text] [Related]  

  • 3. 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]  

  • 4. 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]  

  • 5. Application of N-palmitoyl-O-phosphocholineserine for diagnosis and assessment of response to treatment in Niemann-Pick type C disease.
    Sidhu R; Kell P; Dietzen DJ; Farhat NY; Do AND; Porter FD; Berry-Kravis E; Vite CH; Reunert J; Marquardt T; Giugliani R; Lourenço CM; Bodamer O; Wang RY; Plummer E; Schaffer JE; Ory DS; Jiang X
    Mol Genet Metab; 2020 Apr; 129(4):292-302. PubMed ID: 32033912
    [TBL] [Abstract][Full Text] [Related]  

  • 6. 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]  

  • 7. 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]  

  • 8. 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]  

  • 9. 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]  

  • 10. 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]  

  • 11. Lactose-Appended Hydroxypropyl-β-Cyclodextrin Lowers Cholesterol Accumulation and Alleviates Motor Dysfunction in Niemann-Pick Type C Disease Model Mice.
    Nishida T; Yokoyama R; Kubohira Y; Maeda Y; Takeo T; Nakagata N; Takagi H; Ishikura K; Yanagihara K; Misumi S; Kishimoto N; Ishitsuka Y; Kondo Y; Irie T; Soga M; Era T; Onodera R; Higashi T; Motoyama K
    ACS Appl Bio Mater; 2022 May; 5(5):2377-2388. PubMed ID: 35506864
    [TBL] [Abstract][Full Text] [Related]  

  • 12. 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]  

  • 13. Application of a simple methodology to analyze Hydroxypropyl-β-Cyclodextrin in urine using HPLC-LS in early Niemann-Pick disease type C patient.
    Matencio A; Alcaráz-Gómez MA; García-Carmona F; Arias B; López-Nicolás JM
    J Chromatogr B Analyt Technol Biomed Life Sci; 2018 Sep; 1093-1094():47-51. PubMed ID: 29990712
    [TBL] [Abstract][Full Text] [Related]  

  • 14. Polyrotaxane-based systemic delivery of β-cyclodextrins for potentiating therapeutic efficacy in a mouse model of Niemann-Pick type C disease.
    Tamura A; Yui N
    J Control Release; 2018 Jan; 269():148-158. PubMed ID: 29138063
    [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. 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]  

  • 17. Synthesis of the Anionic Hydroxypropyl-β-cyclodextrin:Poly(decamethylenephosphate) Polyrotaxane and Evaluation of its Cholesterol Efflux Potential in Niemann-Pick C1 Cells.
    Egele K; Samaddar S; Schneider N; Thompson D; Wenz G
    J Mater Chem B; 2019 Jan; 7(4):528-537. PubMed ID: 31372225
    [TBL] [Abstract][Full Text] [Related]  

  • 18. 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]  

  • 19. 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]  

  • 20. Investigation of 2-Hydroxypropyl-β-Cyclodextrin Treatment in a Neuronal-Like Cell Model of Niemann-Pick Type C Using Quantitative Proteomics.
    Cougnoux A; Pergande MR; Serna-Perez F; Cologna SM
    J Am Soc Mass Spectrom; 2023 Apr; 34(4):668-675. PubMed ID: 36920149
    [TBL] [Abstract][Full Text] [Related]  

    [Next]    [New Search]
    of 15.