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267 related items for PubMed ID: 23013746

  • 1. Effect of enzyme therapy and prognostic factors in 69 adults with Pompe disease: an open-label single-center study.
    de Vries JM, van der Beek NA, Hop WC, Karstens FP, Wokke JH, de Visser M, van Engelen BG, Kuks JB, van der Kooi AJ, Notermans NC, Faber CG, Verschuuren JJ, Kruijshaar ME, Reuser AJ, van Doorn PA, van der Ploeg AT.
    Orphanet J Rare Dis; 2012 Sep 26; 7():73. PubMed ID: 23013746
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  • 2. Long-term benefit of enzyme replacement therapy in Pompe disease: A 5-year prospective study.
    Kuperus E, Kruijshaar ME, Wens SCA, de Vries JM, Favejee MM, van der Meijden JC, Rizopoulos D, Brusse E, van Doorn PA, van der Ploeg AT, van der Beek NAME.
    Neurology; 2017 Dec 05; 89(23):2365-2373. PubMed ID: 29117951
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  • 4. Respiratory function during enzyme replacement therapy in late-onset Pompe disease: longitudinal course, prognostic factors, and the impact of time from diagnosis to treatment start.
    Stockton DW, Kishnani P, van der Ploeg A, Llerena J, Boentert M, Roberts M, Byrne BJ, Araujo R, Maruti SS, Thibault N, Verhulst K, Berger KI.
    J Neurol; 2020 Oct 05; 267(10):3038-3053. PubMed ID: 32524257
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  • 9. Clinical features and predictors for disease natural progression in adults with Pompe disease: a nationwide prospective observational study.
    van der Beek NA, de Vries JM, Hagemans ML, Hop WC, Kroos MA, Wokke JH, de Visser M, van Engelen BG, Kuks JB, van der Kooi AJ, Notermans NC, Faber KG, Verschuuren JJ, Reuser AJ, van der Ploeg AT, van Doorn PA.
    Orphanet J Rare Dis; 2012 Nov 12; 7():88. PubMed ID: 23147228
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  • 11. 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 12; 25(4):321-32. PubMed ID: 25617983
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  • 13. The ACE I/D polymorphism does not explain heterogeneity of natural course and response to enzyme replacement therapy in Pompe disease.
    Kuperus E, van der Meijden JC, In 't Groen SLM, Kroos MA, Hoogeveen-Westerveld M, Rizopoulos D, Martinez MYN, Kruijshaar ME, van Doorn PA, van der Beek NAME, van der Ploeg AT, Pijnappel WWMP.
    PLoS One; 2018 Apr 12; 13(12):e0208854. PubMed ID: 30532252
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  • 14. 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 12; 43(6):1219-1231. PubMed ID: 32515844
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  • 18. STIG study: real-world data of long-term outcomes of adults with Pompe disease under enzyme replacement therapy with alglucosidase alfa.
    Gutschmidt K, Musumeci O, Díaz-Manera J, Chien YH, Knop KC, Wenninger S, Montagnese F, Pugliese A, Tavilla G, Alonso-Pérez J, Hwu PW, Toscano A, Schoser B.
    J Neurol; 2021 Jul 12; 268(7):2482-2492. PubMed ID: 33543425
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  • 19. 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 12; 264(4):621-630. PubMed ID: 27372449
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  • 20. 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 12; 101(4):338-45. PubMed ID: 20826098
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