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Journal Abstract Search

521 related items for PubMed ID: 21477922

  • 1. 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; 113(5):350-7. PubMed ID: 21477922
    [Abstract] [Full Text] [Related]

  • 2. Adult Pompe disease: clinical manifestations and outcome of the first Greek patients receiving enzyme replacement therapy.
    Papadimas GK, Spengos K, Konstantinopoulou A, Vassilopoulou S, Vontzalidis A, Papadopoulos C, Michelakakis H, Manta P.
    Clin Neurol Neurosurg; 2011 May; 113(4):303-7. PubMed ID: 21216089
    [Abstract] [Full Text] [Related]

  • 3. Enzyme replacement therapy in severe adult-onset glycogen storage disease type II.
    Ravaglia S, Danesino C, Pichiecchio A, Repetto A, Poloni GU, Rossi M, Fratino P, Moglia A, Costa A.
    Adv Ther; 2008 Aug; 25(8):820-9. PubMed ID: 18704279
    [Abstract] [Full Text] [Related]

  • 4. 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; 101(4):338-45. PubMed ID: 20826098
    [Abstract] [Full Text] [Related]

  • 5. Making diagnosis of Pompe disease at a presymptomatic stage: to treat or not to treat?
    Laloui K, Wary C, Carlier RY, Hogrel JY, Caillaud C, Laforêt P.
    Neurology; 2011 Aug 09; 77(6):594-5. PubMed ID: 21753173
    [No Abstract] [Full Text] [Related]

  • 6. 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 09; 25(4):321-32. PubMed ID: 25617983
    [Abstract] [Full Text] [Related]

  • 7. Recombinant human acid alpha-glucosidase (rhGAA) in adult patients with severe respiratory failure due to Pompe disease.
    Orlikowski D, Pellegrini N, Prigent H, Laforêt P, Carlier R, Carlier P, Eymard B, Lofaso F, Annane D.
    Neuromuscul Disord; 2011 Jul 09; 21(7):477-82. PubMed ID: 21550241
    [Abstract] [Full Text] [Related]

  • 8. Effects of short-to-long term enzyme replacement therapy (ERT) on skeletal muscle tissue in late onset Pompe disease (LOPD).
    Ripolone M, Violano R, Ronchi D, Mondello S, Nascimbeni A, Colombo I, Fagiolari G, Bordoni A, Fortunato F, Lucchini V, Saredi S, Filosto M, Musumeci O, Tonin P, Mongini T, Previtali S, Morandi L, Angelini C, Mora M, Sandri M, Sciacco M, Toscano A, Comi GP, Moggio M.
    Neuropathol Appl Neurobiol; 2018 Aug 09; 44(5):449-462. PubMed ID: 28574618
    [Abstract] [Full Text] [Related]

  • 9. 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 09; 119(1-2):115-23. PubMed ID: 27473031
    [Abstract] [Full Text] [Related]

  • 10. Alglucosidase alfa: new drug. Pompe disease: a short-term benefit.
    Prescrire Int; 2007 Dec 09; 16(92):240-1. PubMed ID: 18092404
    [Abstract] [Full Text] [Related]

  • 11. 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 09; 34(10):834-9. PubMed ID: 22521436
    [Abstract] [Full Text] [Related]

  • 12. Enzyme replacement therapy with alglucosidase alfa in 44 patients with late-onset glycogen storage disease type 2: 12-month results of an observational clinical trial.
    Strothotte S, Strigl-Pill N, Grunert B, Kornblum C, Eger K, Wessig C, Deschauer M, Breunig F, Glocker FX, Vielhaber S, Brejova A, Hilz M, Reiners K, Müller-Felber W, Mengel E, Spranger M, Schoser B.
    J Neurol; 2010 Jan 09; 257(1):91-7. PubMed ID: 19649685
    [Abstract] [Full Text] [Related]

  • 13. Eight years experience with enzyme replacement therapy in two children and one adult with Pompe disease.
    van Capelle CI, Winkel LP, Hagemans ML, Shapira SK, Arts WF, van Doorn PA, Hop WC, Reuser AJ, van der Ploeg AT.
    Neuromuscul Disord; 2008 Jun 09; 18(6):447-52. PubMed ID: 18508267
    [Abstract] [Full Text] [Related]

  • 14. New therapeutic approaches for Pompe disease: enzyme replacement therapy and beyond.
    Kishnani PS, Beckemeyer AA.
    Pediatr Endocrinol Rev; 2014 Sep 09; 12 Suppl 1():114-24. PubMed ID: 25345093
    [Abstract] [Full Text] [Related]

  • 15. 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]

  • 16. Late form of Pompe disease with glycogen storage in peripheral nerves axons.
    Fidziańska A, Ługowska A, Tylki-Szymańska A.
    J Neurol Sci; 2011 Feb 15; 301(1-2):59-62. PubMed ID: 21109266
    [Abstract] [Full Text] [Related]

  • 17. 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]

  • 18. Effects of exercise training during infusion on late-onset Pompe disease patients receiving enzyme replacement therapy.
    Terzis G, Krase A, Papadimas G, Papadopoulos C, Kavouras SA, Manta P.
    Mol Genet Metab; 2012 Dec 24; 107(4):669-73. PubMed ID: 23146291
    [Abstract] [Full Text] [Related]

  • 19. Pompe disease: dramatic improvement in gastrointestinal function following enzyme replacement therapy. A report of three later-onset patients.
    Bernstein DL, Bialer MG, Mehta L, Desnick RJ.
    Mol Genet Metab; 2010 Dec 24; 101(2-3):130-3. PubMed ID: 20638881
    [Abstract] [Full Text] [Related]

  • 20. The emerging phenotype of late-onset Pompe disease: A systematic literature review.
    Chan J, Desai AK, Kazi ZB, Corey K, Austin S, Hobson-Webb LD, Case LE, Jones HN, Kishnani PS.
    Mol Genet Metab; 2017 Mar 24; 120(3):163-172. PubMed ID: 28185884
    [Abstract] [Full Text] [Related]

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