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Journal Abstract Search

579 related items for PubMed ID: 28838325

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  • 2. 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; 360(2):313-323. PubMed ID: 27856936
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  • 6. 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; 20(12):1027-1037. PubMed ID: 34800400
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  • 7. 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(10):1284-1294. PubMed ID: 29565424
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  • 9. 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
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  • 13. Long-term exposure to Myozyme results in a decrease of anti-drug antibodies in late-onset Pompe disease patients.
    Masat E, Laforêt P, De Antonio M, Corre G, Perniconi B, Taouagh N, Mariampillai K, Amelin D, Mauhin W, Hogrel JY, Caillaud C, Ronzitti G, Puzzo F, Kuranda K, Colella P, Mallone R, Benveniste O, Mingozzi F, French Pompe Registry Study Group.
    Sci Rep; 2016 Nov 04; 6():36182. PubMed ID: 27812025
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  • 14. 24-months results in two adults with Pompe disease on enzyme replacement therapy.
    Vielhaber S, Brejova A, Debska-Vielhaber G, Kaufmann J, Feistner H, Schoenfeld MA, Awiszus F.
    Clin Neurol Neurosurg; 2011 Jun 04; 113(5):350-7. PubMed ID: 21477922
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  • 17. Assessing the Role of Anti rh-GAA in Modulating Response to ERT in a Late-Onset Pompe Disease Cohort from the Italian GSDII Study Group.
    Filosto M, Cotti Piccinelli S, Ravaglia S, Servidei S, Moggio M, Musumeci O, Donati MA, Pegoraro E, Di Muzio A, Maggi L, Tonin P, Marrosu G, Sancricca C, Lerario A, Sacchini M, Semplicini C, Bozzoni V, Telese R, Bonanno S, Piras R, Maioli MA, Ricci G, Vercelli L, Galvagni A, Gallo Cassarino S, Caria F, Mongini T, Siciliano G, Padovani A, Toscano A.
    Adv Ther; 2019 May 04; 36(5):1177-1189. PubMed ID: 30879255
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  • 20. Effect of enzyme replacement therapy with alglucosidase alfa (Myozyme®) in 12 patients with advanced late-onset Pompe disease.
    Papadopoulos C, Orlikowski D, Prigent H, Lacour A, Tard C, Furby A, Praline J, Solé G, Hogrel JY, De Antonio M, Semplicini C, Deibener-Kaminsky J, Kaminsky P, Eymard B, Taouagh N, Perniconi B, Hamroun D, Laforêt P, French Pompe Study Group.
    Mol Genet Metab; 2017 Sep 04; 122(1-2):80-85. PubMed ID: 28648663
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