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Journal Abstract Search

682 related items for PubMed ID: 17151339

  • 1. 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
    [Abstract] [Full Text] [Related]

  • 2. 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 09; 149(1):89-97. PubMed ID: 16860134
    [Abstract] [Full Text] [Related]

  • 3. 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]

  • 4. 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]

  • 5. 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 20; 113(5):e448-57. PubMed ID: 15121988
    [Abstract] [Full Text] [Related]

  • 6. Safety and efficacy of recombinant acid alpha-glucosidase (rhGAA) in patients with classical infantile Pompe disease: results of a phase II clinical trial.
    Klinge L, Straub V, Neudorf U, Schaper J, Bosbach T, Görlinger K, Wallot M, Richards S, Voit T.
    Neuromuscul Disord; 2005 Jan 20; 15(1):24-31. PubMed ID: 15639117
    [Abstract] [Full Text] [Related]

  • 7. Pompe disease: early diagnosis and early treatment make a difference.
    Chien YH, Hwu WL, Lee NC.
    Pediatr Neonatol; 2013 Aug 20; 54(4):219-27. PubMed ID: 23632029
    [Abstract] [Full Text] [Related]

  • 8. Enzyme replacement therapy in classical infantile pompe disease: results of a ten-month follow-up study.
    Klinge L, Straub V, Neudorf U, Voit T.
    Neuropediatrics; 2005 Feb 20; 36(1):6-11. PubMed ID: 15776317
    [Abstract] [Full Text] [Related]

  • 9. A new assay for fast, reliable CRIM status determination in infantile-onset Pompe disease.
    Wang Z, Okamoto P, Keutzer J.
    Mol Genet Metab; 2014 Feb 20; 111(2):92-100. PubMed ID: 24044919
    [Abstract] [Full Text] [Related]

  • 10. A study on the safety and efficacy of reveglucosidase alfa in patients with late-onset Pompe disease.
    Byrne BJ, Geberhiwot T, Barshop BA, Barohn R, Hughes D, Bratkovic D, Desnuelle C, Laforet P, Mengel E, Roberts M, Haroldsen P, Reilley K, Jayaram K, Yang K, Walsh L, POM-001/002 Investigators.
    Orphanet J Rare Dis; 2017 Aug 24; 12(1):144. PubMed ID: 28838325
    [Abstract] [Full Text] [Related]

  • 11. Effect of alglucosidase alfa dosage on survival and walking ability in patients with classic infantile Pompe disease: a multicentre observational cohort study from the European Pompe Consortium.
    Ditters IAM, Huidekoper HH, Kruijshaar ME, Rizopoulos D, Hahn A, Mongini TE, Labarthe F, Tardieu M, Chabrol B, Brassier A, Parini R, Parenti G, van der Beek NAME, van der Ploeg AT, van den Hout JMP, European Pompe Consortium project group on classic infantile Pompe disease.
    Lancet Child Adolesc Health; 2022 Jan 24; 6(1):28-37. PubMed ID: 34822769
    [Abstract] [Full Text] [Related]

  • 12. Long-term prognosis of patients with infantile-onset Pompe disease diagnosed by newborn screening and treated since birth.
    Chien YH, Lee NC, Chen CA, Tsai FJ, Tsai WH, Shieh JY, Huang HJ, Hsu WC, Tsai TH, Hwu WL.
    J Pediatr; 2015 Apr 24; 166(4):985-91.e1-2. PubMed ID: 25466677
    [Abstract] [Full Text] [Related]

  • 13. The generation of induced pluripotent stem cells (iPSCs) from patients with infantile and late-onset types of Pompe disease and the effects of treatment with acid-α-glucosidase in Pompe's iPSCs.
    Higuchi T, Kawagoe S, Otsu M, Shimada Y, Kobayashi H, Hirayama R, Eto K, Ida H, Ohashi T, Nakauchi H, Eto Y.
    Mol Genet Metab; 2014 May 24; 112(1):44-8. PubMed ID: 24642446
    [Abstract] [Full Text] [Related]

  • 14. [Clinical sequelae of 17 cases with glycogen storage disease type II/Pompe disease].
    Zhang HB, Zhang WM, Qiu JJ, Meng Y, Qiu ZQ.
    Zhonghua Er Ke Za Zhi; 2012 Jun 24; 50(6):415-9. PubMed ID: 22931935
    [Abstract] [Full Text] [Related]

  • 15. Hyaluronidase increases the biodistribution of acid alpha-1,4 glucosidase in the muscle of Pompe disease mice: an approach to enhance the efficacy of enzyme replacement therapy.
    Matalon R, Surendran S, Campbell GA, Michals-Matalon K, Tyring SK, Grady J, Cheng S, Kaye E.
    Biochem Biophys Res Commun; 2006 Nov 24; 350(3):783-7. PubMed ID: 17027913
    [Abstract] [Full Text] [Related]

  • 16. Enzyme replacement therapy and immunotherapy lead to significant functional improvement in two children with Pompe disease: a case report.
    Castellar-Leones SM, Ortiz-Corredor F, Manrique-Hernández D, Sánchez-Peñarete D, Ruiz-Ospina E, Soto-Peña D, Correa-Arrieta C.
    J Med Case Rep; 2024 Jul 18; 18(1):328. PubMed ID: 39020349
    [Abstract] [Full Text] [Related]

  • 17. Report of the first Brazilian infantile Pompe disease patient to be treated with recombinant human acid alpha-glucosidase.
    Pereira SJ, Berditchevisky CR, Marie SK.
    J Pediatr (Rio J); 2008 Jul 18; 84(3):272-5. PubMed ID: 18535739
    [Abstract] [Full Text] [Related]

  • 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 18; 27(3):319-24. PubMed ID: 21940687
    [Abstract] [Full Text] [Related]

  • 19. 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]

  • 20. The role of immune tolerance induction in restoration of the efficacy of ERT in Pompe disease.
    Lacaná E, Yao LP, Pariser AR, Rosenberg AS.
    Am J Med Genet C Semin Med Genet; 2012 Feb 15; 160C(1):30-9. PubMed ID: 22253234
    [Abstract] [Full Text] [Related]

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