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Journal Abstract Search

350 related items for PubMed ID: 19287243

  • 1. Clinical outcomes after long-term treatment with alglucosidase alfa in infants and children with advanced Pompe disease.
    Nicolino M, Byrne B, Wraith JE, Leslie N, Mandel H, Freyer DR, Arnold GL, Pivnick EK, Ottinger CJ, Robinson PH, Loo JC, Smitka M, Jardine P, Tatò L, Chabrol B, McCandless S, Kimura S, Mehta L, Bali D, Skrinar A, Morgan C, Rangachari L, Corzo D, Kishnani PS.
    Genet Med; 2009 Mar; 11(3):210-9. PubMed ID: 19287243
    [Abstract] [Full Text] [Related]

  • 2. Enzyme replacement therapy for infantile-onset Pompe disease.
    Chen M, Zhang L, Quan S.
    Cochrane Database Syst Rev; 2017 Nov 20; 11(11):CD011539. PubMed ID: 29155436
    [Abstract] [Full Text] [Related]

  • 3. 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 20; 20(10):1284-1294. PubMed ID: 29565424
    [Abstract] [Full Text] [Related]

  • 4. 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 20; 29(3):167-186. PubMed ID: 30770310
    [Abstract] [Full Text] [Related]

  • 5. A randomized study of alglucosidase alfa in late-onset Pompe's disease.
    van der Ploeg AT, Clemens PR, Corzo D, Escolar DM, Florence J, Groeneveld GJ, Herson S, Kishnani PS, Laforet P, Lake SL, Lange DJ, Leshner RT, Mayhew JE, Morgan C, Nozaki K, Park DJ, Pestronk A, Rosenbloom B, Skrinar A, van Capelle CI, van der Beek NA, Wasserstein M, Zivkovic SA.
    N Engl J Med; 2010 Apr 15; 362(15):1396-406. PubMed ID: 20393176
    [Abstract] [Full Text] [Related]

  • 6. Safety and efficacy of avalglucosidase alfa versus alglucosidase alfa in patients with late-onset Pompe disease (COMET): a phase 3, randomised, multicentre trial.
    Diaz-Manera J, Kishnani PS, Kushlaf H, Ladha S, Mozaffar T, Straub V, Toscano A, van der Ploeg AT, Berger KI, Clemens PR, Chien YH, Day JW, Illarioshkin S, Roberts M, Attarian S, Borges JL, Bouhour F, Choi YC, Erdem-Ozdamar S, Goker-Alpan O, Kostera-Pruszczyk A, Haack KA, Hug C, Huynh-Ba O, Johnson J, Thibault N, Zhou T, Dimachkie MM, Schoser B, COMET Investigator Group.
    Lancet Neurol; 2021 Dec 15; 20(12):1012-1026. PubMed ID: 34800399
    [Abstract] [Full Text] [Related]

  • 7. 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
    [Abstract] [Full Text] [Related]

  • 8. Alglucosidase alfa: new drug. Pompe disease: a short-term benefit.
    Prescrire Int; 2007 Dec 15; 16(92):240-1. PubMed ID: 18092404
    [Abstract] [Full Text] [Related]

  • 9. An individually, modified approach to desensitize infants and young children with Pompe disease, and significant reactions to alglucosidase alfa infusions.
    El-Gharbawy AH, Mackey J, DeArmey S, Westby G, Grinnell SG, Malovrh P, Conway R, Kishnani PS.
    Mol Genet Metab; 2011 Dec 15; 104(1-2):118-22. PubMed ID: 21802969
    [Abstract] [Full Text] [Related]

  • 10. Early treatment with alglucosidase alpha prolongs long-term survival of infants with Pompe disease.
    Kishnani PS, Corzo D, Leslie ND, Gruskin D, Van der Ploeg A, Clancy JP, Parini R, Morin G, Beck M, Bauer MS, Jokic M, Tsai CE, Tsai BW, Morgan C, O'Meara T, Richards S, Tsao EC, Mandel H.
    Pediatr Res; 2009 Sep 15; 66(3):329-35. PubMed ID: 19542901
    [Abstract] [Full Text] [Related]

  • 11. Safety and efficacy of cipaglucosidase alfa plus miglustat versus alglucosidase alfa plus placebo in late-onset Pompe disease (PROPEL): an international, randomised, double-blind, parallel-group, phase 3 trial.
    Schoser B, Roberts M, Byrne BJ, Sitaraman S, Jiang H, Laforêt P, Toscano A, Castelli J, Díaz-Manera J, Goldman M, van der Ploeg AT, Bratkovic D, Kuchipudi S, Mozaffar T, Kishnani PS, PROPEL Study Group.
    Lancet Neurol; 2021 Dec 15; 20(12):1027-1037. PubMed ID: 34800400
    [Abstract] [Full Text] [Related]

  • 12. 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
    [Abstract] [Full Text] [Related]

  • 13. Long-term intravenous treatment of Pompe disease with recombinant human alpha-glucosidase from milk.
    Van den Hout JM, Kamphoven JH, Winkel LP, Arts WF, De Klerk JB, Loonen MC, Vulto AG, Cromme-Dijkhuis A, Weisglas-Kuperus N, Hop W, Van Hirtum H, Van Diggelen OP, Boer M, Kroos MA, Van Doorn PA, Van der Voort E, Sibbles B, Van Corven EJ, Brakenhoff JP, Van Hove J, Smeitink JA, de Jong G, Reuser AJ, Van der Ploeg AT.
    Pediatrics; 2004 May 15; 113(5):e448-57. PubMed ID: 15121988
    [Abstract] [Full Text] [Related]

  • 14. Effects of a higher dose of alglucosidase alfa on ventilator-free survival and motor outcome in classic infantile Pompe disease: an open-label single-center study.
    van Gelder CM, Poelman E, Plug I, Hoogeveen-Westerveld M, van der Beek NAME, Reuser AJJ, van der Ploeg AT.
    J Inherit Metab Dis; 2016 May 15; 39(3):383-390. PubMed ID: 26768149
    [Abstract] [Full Text] [Related]

  • 15. Survival and long-term outcomes in late-onset Pompe disease following alglucosidase alfa treatment: a systematic review and meta-analysis.
    Schoser B, Stewart A, Kanters S, Hamed A, Jansen J, Chan K, Karamouzian M, Toscano A.
    J Neurol; 2017 Apr 15; 264(4):621-630. PubMed ID: 27372449
    [Abstract] [Full Text] [Related]

  • 16. 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
    [Abstract] [Full Text] [Related]

  • 17. 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 15; 98(3):319-21. PubMed ID: 19640753
    [Abstract] [Full Text] [Related]

  • 18. 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 15; 107(3):456-61. PubMed ID: 23031366
    [Abstract] [Full Text] [Related]

  • 19. Chinese hamster ovary cell-derived recombinant human acid alpha-glucosidase in infantile-onset Pompe disease.
    Kishnani PS, Nicolino M, Voit T, Rogers RC, Tsai AC, Waterson J, Herman GE, Amalfitano A, Thurberg BL, Richards S, Davison M, Corzo D, Chen YT.
    J Pediatr; 2006 Jul 15; 149(1):89-97. PubMed ID: 16860134
    [Abstract] [Full Text] [Related]

  • 20. Long-term Safety and Efficacy of Avalglucosidase Alfa in Patients With Late-Onset Pompe Disease.
    Dimachkie MM, Barohn RJ, Byrne B, Goker-Alpan O, Kishnani PS, Ladha S, Laforêt P, Mengel KE, Peña LDM, Sacconi S, Straub V, Trivedi J, Van Damme P, van der Ploeg AT, Vissing J, Young P, Haack KA, Foster M, Gilbert JM, Miossec P, Vitse O, Zhou T, Schoser B, NEO-EXT investigators.
    Neurology; 2022 Aug 01; 99(5):e536-e548. PubMed ID: 35618441
    [Abstract] [Full Text] [Related]

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