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Journal Abstract Search

282 related items for PubMed ID: 22815812

  • 1. The pharmacological chaperone AT2220 increases recombinant human acid α-glucosidase uptake and glycogen reduction in a mouse model of Pompe disease.
    Khanna R, Flanagan JJ, Feng J, Soska R, Frascella M, Pellegrino LJ, Lun Y, Guillen D, Lockhart DJ, Valenzano KJ.
    PLoS One; 2012; 7(7):e40776. PubMed ID: 22815812
    [Abstract] [Full Text] [Related]

  • 2. The pharmacological chaperone AT2220 increases the specific activity and lysosomal delivery of mutant acid alpha-glucosidase, and promotes glycogen reduction in a transgenic mouse model of Pompe disease.
    Khanna R, Powe AC, Lun Y, Soska R, Feng J, Dhulipala R, Frascella M, Garcia A, Pellegrino LJ, Xu S, Brignol N, Toth MJ, Do HV, Lockhart DJ, Wustman BA, Valenzano KJ.
    PLoS One; 2014; 9(7):e102092. PubMed ID: 25036864
    [Abstract] [Full Text] [Related]

  • 3. Duvoglustat HCl Increases Systemic and Tissue Exposure of Active Acid α-Glucosidase in Pompe Patients Co-administered with Alglucosidase α.
    Kishnani P, Tarnopolsky M, Roberts M, Sivakumar K, Dasouki M, Dimachkie MM, Finanger E, Goker-Alpan O, Guter KA, Mozaffar T, Pervaiz MA, Laforet P, Levine T, Adera M, Lazauskas R, Sitaraman S, Khanna R, Benjamin E, Feng J, Flanagan JJ, Barth J, Barlow C, Lockhart DJ, Valenzano KJ, Boudes P, Johnson FK, Byrne B.
    Mol Ther; 2017 May 03; 25(5):1199-1208. PubMed ID: 28341561
    [Abstract] [Full Text] [Related]

  • 4. Antibody-mediated enzyme replacement therapy targeting both lysosomal and cytoplasmic glycogen in Pompe disease.
    Yi H, Sun T, Armstrong D, Borneman S, Yang C, Austin S, Kishnani PS, Sun B.
    J Mol Med (Berl); 2017 May 03; 95(5):513-521. PubMed ID: 28154884
    [Abstract] [Full Text] [Related]

  • 5. Improved efficacy of a next-generation ERT in murine Pompe disease.
    Xu S, Lun Y, Frascella M, Garcia A, Soska R, Nair A, Ponery AS, Schilling A, Feng J, Tuske S, Valle MCD, Martina JA, Ralston E, Gotschall R, Valenzano KJ, Puertollano R, Do HV, Raben N, Khanna R.
    JCI Insight; 2019 Mar 07; 4(5):. PubMed ID: 30843882
    [Abstract] [Full Text] [Related]

  • 6. Carbohydrate-remodelled acid alpha-glucosidase with higher affinity for the cation-independent mannose 6-phosphate receptor demonstrates improved delivery to muscles of Pompe mice.
    Zhu Y, Li X, McVie-Wylie A, Jiang C, Thurberg BL, Raben N, Mattaliano RJ, Cheng SH.
    Biochem J; 2005 Aug 01; 389(Pt 3):619-28. PubMed ID: 15839836
    [Abstract] [Full Text] [Related]

  • 7. Reveglucosidase alfa (BMN 701), an IGF2-Tagged rhAcid α-Glucosidase, Improves Respiratory Functional Parameters in a Murine Model of Pompe Disease.
    Peng J, Dalton J, Butt M, Tracy K, Kennedy D, Haroldsen P, Cahayag R, Zoog S, O'Neill CA, Tsuruda LS.
    J Pharmacol Exp Ther; 2017 Feb 01; 360(2):313-323. PubMed ID: 27856936
    [Abstract] [Full Text] [Related]

  • 8. Efficient therapy for refractory Pompe disease by mannose 6-phosphate analogue grafting on acid α-glucosidase.
    Basile I, Da Silva A, El Cheikh K, Godefroy A, Daurat M, Harmois A, Perez M, Caillaud C, Charbonné HV, Pau B, Gary-Bobo M, Morère A, Garcia M, Maynadier M.
    J Control Release; 2018 Jan 10; 269():15-23. PubMed ID: 29108866
    [Abstract] [Full Text] [Related]

  • 9. Increasing Enzyme Mannose-6-Phosphate Levels but Not Miglustat Coadministration Enhances the Efficacy of Enzyme Replacement Therapy in Pompe Mice.
    Anding A, Kinton S, Baranowski K, Brezzani A, De Busser H, Dufault MR, Finn P, Keefe K, Tetrault T, Li Y, Qiu W, Raes K, Vitse O, Zhang M, Ziegler R, Sardi SP, Hunter B, George K.
    J Pharmacol Exp Ther; 2023 Nov 10; 387(2):188-203. PubMed ID: 37679046
    [Abstract] [Full Text] [Related]

  • 10. Biochemical and pharmacological characterization of different recombinant acid alpha-glucosidase preparations evaluated for the treatment of Pompe disease.
    McVie-Wylie AJ, Lee KL, Qiu H, Jin X, Do H, Gotschall R, Thurberg BL, Rogers C, Raben N, O'Callaghan M, Canfield W, Andrews L, McPherson JM, Mattaliano RJ.
    Mol Genet Metab; 2008 Aug 10; 94(4):448-455. PubMed ID: 18538603
    [Abstract] [Full Text] [Related]

  • 11. Pharmacological enhancement of α-glucosidase by the allosteric chaperone N-acetylcysteine.
    Porto C, Ferrara MC, Meli M, Acampora E, Avolio V, Rosa M, Cobucci-Ponzano B, Colombo G, Moracci M, Andria G, Parenti G.
    Mol Ther; 2012 Dec 10; 20(12):2201-11. PubMed ID: 22990675
    [Abstract] [Full Text] [Related]

  • 12. Conjugation of mannose 6-phosphate-containing oligosaccharides to acid alpha-glucosidase improves the clearance of glycogen in pompe mice.
    Zhu Y, Li X, Kyazike J, Zhou Q, Thurberg BL, Raben N, Mattaliano RJ, Cheng SH.
    J Biol Chem; 2004 Nov 26; 279(48):50336-41. PubMed ID: 15383547
    [Abstract] [Full Text] [Related]

  • 13. Glycosylation-independent lysosomal targeting of acid α-glucosidase enhances muscle glycogen clearance in pompe mice.
    Maga JA, Zhou J, Kambampati R, Peng S, Wang X, Bohnsack RN, Thomm A, Golata S, Tom P, Dahms NM, Byrne BJ, LeBowitz JH.
    J Biol Chem; 2013 Jan 18; 288(3):1428-38. PubMed ID: 23188827
    [Abstract] [Full Text] [Related]

  • 14. The Release of a Soluble Glycosylated Protein from Glycogen by Recombinant Lysosomal α-Glucosidase (rhGAA) In Vitro and Its Presence in Serum In Vivo.
    Murray AK.
    Biomolecules; 2020 Nov 29; 10(12):. PubMed ID: 33260301
    [Abstract] [Full Text] [Related]

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  • 17. A beta-blocker, propranolol, decreases the efficacy from enzyme replacement therapy in Pompe disease.
    Han SO, Pope R, Li S, Kishnani PS, Steet R, Koeberl DD.
    Mol Genet Metab; 2016 Feb 29; 117(2):114-9. PubMed ID: 26454691
    [Abstract] [Full Text] [Related]

  • 18. Replacing acid alpha-glucosidase in Pompe disease: recombinant and transgenic enzymes are equipotent, but neither completely clears glycogen from type II muscle fibers.
    Raben N, Fukuda T, Gilbert AL, de Jong D, Thurberg BL, Mattaliano RJ, Meikle P, Hopwood JJ, Nagashima K, Nagaraju K, Plotz PH.
    Mol Ther; 2005 Jan 29; 11(1):48-56. PubMed ID: 15585405
    [Abstract] [Full Text] [Related]

  • 19. Pharmacological Chaperone Therapy for Pompe Disease.
    Borie-Guichot M, Tran ML, Génisson Y, Ballereau S, Dehoux C.
    Molecules; 2021 Nov 29; 26(23):. PubMed ID: 34885805
    [Abstract] [Full Text] [Related]

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