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Journal Abstract Search

179 related items for PubMed ID: 30778879

  • 1. Desensitization of two young patients with infantile-onset Pompe disease and severe reactions to alglucosidase alfa.
    Gragnaniello V, Fecarotta S, Pecoraro A, Tarallo A, Catzola A, Spadaro G, Parenti G, Della Casa R.
    Neurol Sci; 2019 Jul; 40(7):1453-1455. PubMed ID: 30778879
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  • 7. First successful concomitant therapy of immune tolerance induction therapy and desensitization in a CRIM-negative infantile Pompe patient.
    Emecen Sanli M, Ertoy Karagol HI, Kilic A, Aktasoglu E, Inci A, Okur I, Ezgu F, Tumer L.
    J Pediatr Endocrinol Metab; 2022 Feb 23; 35(2):273-277. PubMed ID: 34561975
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  • 8. Hypersensitivity infusion-associated reactions induced by enzyme replacement therapy in a cohort of patients with late-onset Pompe disease: An experience from the French Pompe Registry.
    Lessard LER, Tard C, Salort-Campana E, Sacconi S, Béhin A, Bassez G, Orlikowski D, Merle P, Nollet S, Gallay L, Bérard F, Robinson P, Bouhour F, Laforêt P.
    Mol Genet Metab; 2023 Jul 23; 139(3):107611. PubMed ID: 37285781
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  • 10. Desensitization of an adult patient with Pompe disease and a history of anaphylaxis to alglucosidase alfa.
    Lipinski SE, Lipinski MJ, Burnette A, Platts-Mills TA, Wilson WG.
    Mol Genet Metab; 2009 Nov 23; 98(3):319-21. PubMed ID: 19640753
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  • 11. Efficacy, safety profile, and immunogenicity of alglucosidase alfa produced at the 4,000-liter scale in US children and adolescents with Pompe disease: ADVANCE, a phase IV, open-label, prospective study.
    Hahn SH, Kronn D, Leslie ND, Pena LDM, Tanpaiboon P, Gambello MJ, Gibson JB, Hillman R, Stockton DW, Day JW, Wang RY, An Haack K, Shafi R, Sparks S, Zhao Y, Wilson C, Kishnani PS, Pompe ADVANCE Study Consortium.
    Genet Med; 2018 Oct 23; 20(10):1284-1294. PubMed ID: 29565424
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  • 12. Improvement with ongoing Enzyme Replacement Therapy in advanced late-onset Pompe disease: a case study.
    Case LE, Koeberl DD, Young SP, Bali D, DeArmey SM, Mackey J, Kishnani PS.
    Mol Genet Metab; 2008 Dec 23; 95(4):233-5. PubMed ID: 18930676
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  • 14. Safety and efficacy of alternative alglucosidase alfa regimens in Pompe disease.
    Case LE, Bjartmar C, Morgan C, Casey R, Charrow J, Clancy JP, Dasouki M, DeArmey S, Nedd K, Nevins M, Peters H, Phillips D, Spigelman Z, Tifft C, Kishnani PS.
    Neuromuscul Disord; 2015 Apr 23; 25(4):321-32. PubMed ID: 25617983
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  • 19. Pompe disease: early diagnosis and early treatment make a difference.
    Chien YH, Hwu WL, Lee NC.
    Pediatr Neonatol; 2013 Aug 23; 54(4):219-27. PubMed ID: 23632029
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  • 20. Recombinant human acid [alpha]-glucosidase: major clinical benefits in infantile-onset Pompe disease.
    Kishnani PS, Corzo D, Nicolino M, Byrne B, Mandel H, Hwu WL, Leslie N, Levine J, Spencer C, McDonald M, Li J, Dumontier J, Halberthal M, Chien YH, Hopkin R, Vijayaraghavan S, Gruskin D, Bartholomew D, van der Ploeg A, Clancy JP, Parini R, Morin G, Beck M, De la Gastine GS, Jokic M, Thurberg B, Richards S, Bali D, Davison M, Worden MA, Chen YT, Wraith JE.
    Neurology; 2007 Jan 09; 68(2):99-109. PubMed ID: 17151339
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