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Journal Abstract Search

224 related items for PubMed ID: 33851281

  • 21. Cardiopulmonary exercise test to quantify enzyme replacement response in pediatric Pompe disease.
    Bar-Yoseph R, Mandel H, Mainzer G, Gur M, Tal G, Shalloufeh G, Bentur L.
    Pediatr Pulmonol; 2018 Mar; 53(3):366-373. PubMed ID: 29356433
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  • 22. Observational clinical study in juvenile-adult glycogenosis type 2 patients undergoing enzyme replacement therapy for up to 4 years.
    Angelini C, Semplicini C, Ravaglia S, Bembi B, Servidei S, Pegoraro E, Moggio M, Filosto M, Sette E, Crescimanno G, Tonin P, Parini R, Morandi L, Marrosu G, Greco G, Musumeci O, Di Iorio G, Siciliano G, Donati MA, Carubbi F, Ermani M, Mongini T, Toscano A, Italian GSDII Group.
    J Neurol; 2012 May; 259(5):952-8. PubMed ID: 22081099
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  • 23. A Systematic Review and Meta-Analysis of Enzyme Replacement Therapy in Late-Onset Pompe Disease.
    Dornelles AD, Junges APP, Pereira TV, Krug BC, Gonçalves CBT, Llerena JC, Kishnani PS, de Oliveira HA, Schwartz IVD.
    J Clin Med; 2021 Oct 21; 10(21):. PubMed ID: 34768348
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  • 24. 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 21; 120(3):163-172. PubMed ID: 28185884
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  • 26. Study of the effect of anti-rhGAA antibodies at low and intermediate titers in late onset Pompe patients treated with ERT.
    Fernández-Simón E, Carrasco-Rozas A, Gallardo E, González-Quereda L, Alonso-Pérez J, Belmonte I, Pedrosa-Hernández I, Montiel E, Segovia S, Suárez-Calvet X, Llauger J, Mayos M, Illa I, Barba-Romero MA, Barcena J, Paradas C, Carzorla MR, Creus C, Coll-Cantí J, Díaz M, Domínguez C, Fernández-Torrón R, García-Antelo MJ, Grau JM, López de Munáin A, Martínez-García FA, Morgado Y, Moreno A, Morís G, Muñoz-Blanco MA, Nascimento A, Parajuá-Pozo JL, Querol L, Rojas R, Robledo-Strauss A, Rojas-Marcos Í, Salazar JA, Usón M, Díaz-Manera J.
    Mol Genet Metab; 2019 Mar 21; 128(1-2):129-136. PubMed ID: 31378569
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  • 27. 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
    [Abstract] [Full Text] [Related]

  • 28. White matter lesions in treated late onset Pompe disease are not different to matched controls.
    Schneider I, Hensel O, Zierz S.
    Mol Genet Metab; 2019 Jun 04; 127(2):128-131. PubMed ID: 31153821
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  • 30. Effectiveness of enzyme replacement therapy in adults with late-onset Pompe disease: results from the NCS-LSD cohort study.
    Anderson LJ, Henley W, Wyatt KM, Nikolaou V, Waldek S, Hughes DA, Lachmann RH, Logan S.
    J Inherit Metab Dis; 2014 Nov 04; 37(6):945-52. PubMed ID: 24906254
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  • 31. Real-life effectiveness 1 year after switching to avalglucosidase alfa in late-onset Pompe disease patients worsening on alglucosidase alfa therapy: A French cohort study.
    Tard C, Bouhour F, Michaud M, Beltran S, Fournier M, Demurger F, Lagrange E, Nollet S, Sacconi S, Noury JB, Magot A, Cintas P, Renard D, Deibener-Kaminsky J, Lefeuvre C, Davion JB, Salort-Campana E, Arrassi A, Taouagh N, Spinazzi M, Pompe Study Group, Attarian S, Laforêt P.
    Eur J Neurol; 2024 Jul 04; 31(7):e16292. PubMed ID: 38587143
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  • 34. 104-week efficacy and safety of cipaglucosidase alfa plus miglustat in adults with late-onset Pompe disease: a phase III open-label extension study (ATB200-07).
    Schoser B, Kishnani PS, Bratkovic D, Byrne BJ, Claeys KG, Díaz-Manera J, Laforêt P, Roberts M, Toscano A, van der Ploeg AT, Castelli J, Goldman M, Holdbrook F, Sitaraman Das S, Wasfi Y, Mozaffar T, ATB200-07 Study Group.
    J Neurol; 2024 May 04; 271(5):2810-2823. PubMed ID: 38418563
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  • 38. Comparing the efficacy of cipaglucosidase alfa plus miglustat with other enzyme replacement therapies for late-onset Pompe disease: a network meta-analysis utilizing patient-level and aggregate data.
    Shohet S, Hummel N, Fu S, Keyzor I, MacCulloch A, Johnson N, Castelli J, Czarny-Ozga I, Mozaffar T, Thom H.
    J Comp Eff Res; 2024 Oct 04; 13(10):e240045. PubMed ID: 39287071
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