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Journal Abstract Search

391 related items for PubMed ID: 26279333

  • 1. Long-term enzyme-replacement therapy (ERT) with alglucosidase alfa: Evolution of two siblings with juvenile late-onset Pompe disease.
    Rafael Bretón Martínez J, Martínez AC.
    J Neurol Sci; 2015 Nov 15; 358(1-2):459-60. PubMed ID: 26279333
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  • 2. 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 15; 25(4):321-32. PubMed ID: 25617983
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  • 3. Prospective exploratory muscle biopsy, imaging, and functional assessment in patients with late-onset Pompe disease treated with alglucosidase alfa: The EMBASSY Study.
    van der Ploeg A, Carlier PG, Carlier RY, Kissel JT, Schoser B, Wenninger S, Pestronk A, Barohn RJ, Dimachkie MM, Goker-Alpan O, Mozaffar T, Pena LD, Simmons Z, Straub V, Guglieri M, Young P, Boentert M, Baudin PY, Wens S, Shafi R, Bjartmar C, Thurberg BL.
    Mol Genet Metab; 2016 Sep 15; 119(1-2):115-23. PubMed ID: 27473031
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  • 8. High antibody titer in an adult with Pompe disease affects treatment with alglucosidase alfa.
    de Vries JM, van der Beek NA, Kroos MA, Ozkan L, van Doorn PA, Richards SM, Sung CC, Brugma JD, Zandbergen AA, van der Ploeg AT, Reuser AJ.
    Mol Genet Metab; 2010 Dec 15; 101(4):338-45. PubMed ID: 20826098
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  • 10. Quantitative computed tomography for enzyme replacement therapy in Pompe disease.
    Yonee C, Toyoshima M, Young SP, Maruyama S, Higuchi I, Narita A, Maegaki Y, Nanba E, Ohno K, Kawano Y.
    Brain Dev; 2012 Nov 15; 34(10):834-9. PubMed ID: 22521436
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  • 11. Enzyme replacement therapy with alglucosidase alfa in Pompe disease: Clinical experience with rate escalation.
    Desai AK, Walters CK, Cope HL, Kazi ZB, DeArmey SM, Kishnani PS.
    Mol Genet Metab; 2018 Feb 15; 123(2):92-96. PubMed ID: 29289479
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  • 12. Safety, tolerability, pharmacokinetics, pharmacodynamics, and exploratory efficacy of the novel enzyme replacement therapy avalglucosidase alfa (neoGAA) in treatment-naïve and alglucosidase alfa-treated patients with late-onset Pompe disease: A phase 1, open-label, multicenter, multinational, ascending dose study.
    Pena LDM, Barohn RJ, Byrne BJ, Desnuelle C, Goker-Alpan O, Ladha S, Laforêt P, Mengel KE, Pestronk A, Pouget J, Schoser B, Straub V, Trivedi J, Van Damme P, Vissing J, Young P, Kacena K, Shafi R, Thurberg BL, Culm-Merdek K, van der Ploeg AT, NEO1 Investigator Group.
    Neuromuscul Disord; 2019 Mar 15; 29(3):167-186. PubMed ID: 30770310
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  • 13. 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 15; 95(4):233-5. PubMed ID: 18930676
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  • 14. 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
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  • 17. Open-label extension study following the Late-Onset Treatment Study (LOTS) of alglucosidase alfa.
    van der Ploeg AT, Barohn R, Carlson L, Charrow J, Clemens PR, Hopkin RJ, Kishnani PS, Laforêt P, Morgan C, Nations S, Pestronk A, Plotkin H, Rosenbloom BE, Sims KB, Tsao E.
    Mol Genet Metab; 2012 Nov 07; 107(3):456-61. PubMed ID: 23031366
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  • 18. Infantile Pompe disease: clinical and genetic characteristics with an experience of enzyme replacement therapy.
    Cho A, Kim SJ, Lim BC, Hwang H, Park JD, Kim GB, Jin DK, Lee J, Ki CS, Kim KJ, Hwang YS, Chae JH.
    J Child Neurol; 2012 Mar 07; 27(3):319-24. PubMed ID: 21940687
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  • 19. Unwarranted, long term, alglucosidase alfa enzyme replacement therapy in two non-Pompe disease patients.
    Karam C, Ragole T, Moshe-Lilie O, Chahin N.
    Clin Neurol Neurosurg; 2020 Sep 07; 196():106048. PubMed ID: 32623214
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  • 20. Long-term benefit of enzyme replacement therapy with alglucosidase alfa in adults with Pompe disease: Prospective analysis from the French Pompe Registry.
    Semplicini C, De Antonio M, Taouagh N, Béhin A, Bouhour F, Echaniz-Laguna A, Magot A, Nadaj-Pakleza A, Orlikowski D, Sacconi S, Salort-Campana E, Solé G, Tard C, Zagnoli F, Hogrel JY, Hamroun D, Laforêt P, French Pompe Study Group.
    J Inherit Metab Dis; 2020 Nov 07; 43(6):1219-1231. PubMed ID: 32515844
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