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Journal Abstract Search

132 related items for PubMed ID: 39318718

  • 1. Health-Related Quality-of-Life Utility Values in Adults With Late-Onset Pompe Disease: Analyses of EQ-5D Data From the PROPEL Clinical Trial.
    MacCulloch A, Griffiths A, Johnson N, Shohet S.
    J Health Econ Outcomes Res; 2024; 11(2):80-85. PubMed ID: 39318718
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  • 2. 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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  • 3. Enzyme replacement therapy for late-onset Pompe disease.
    Dalmia S, Sharma R, Ramaswami U, Hughes D, Jahnke N, Cole D, Smith S, Remmington T.
    Cochrane Database Syst Rev; 2023 Dec 12; 12(12):CD012993. PubMed ID: 38084761
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  • 4. Quality of Life with Late-Onset Pompe Disease: Qualitative Interviews and General Public Utility Estimation in the United Kingdom.
    Hubig L, Sussex AK, MacCulloch A, Hughes D, Graham R, Morris L, Raza S, Lloyd AJ, Sowinski A, Gallop K.
    J Health Econ Outcomes Res; 2023 Dec 12; 10(1):41-50. PubMed ID: 36883056
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  • 5. Effect of avalglucosidase alfa on disease-specific and general patient-reported outcomes in treatment-naïve adults with late-onset Pompe disease compared with alglucosidase alfa: Meaningful change analyses from the Phase 3 COMET trial.
    Toscano A, Pollissard L, Msihid J, van der Beek N, Kishnani PS, Dimachkie MM, Berger KI, DasMahapatra P, Thibault N, Hamed A, Zhou T, Haack KA, Schoser B.
    Mol Genet Metab; 2024 Feb 12; 141(2):108121. PubMed ID: 38184428
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  • 6. 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 12; 271(5):2810-2823. PubMed ID: 38418563
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  • 9. Catalog of EQ-5D-3L Health-Related Quality-of-Life Scores for 199 Chronic Conditions and Health Risks in Denmark.
    Hvidberg MF, Petersen KD, Davidsen M, Witt Udsen F, Frølich A, Ehlers L, Alava MH.
    MDM Policy Pract; 2023 May 12; 8(1):23814683231159023. PubMed ID: 37056295
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  • 11. Long-term Safety and Efficacy of Avalglucosidase Alfa in Patients With Late-Onset Pompe Disease.
    Dimachkie MM, Barohn RJ, Byrne B, Goker-Alpan O, Kishnani PS, Ladha S, Laforêt P, Mengel KE, Peña LDM, Sacconi S, Straub V, Trivedi J, Van Damme P, van der Ploeg AT, Vissing J, Young P, Haack KA, Foster M, Gilbert JM, Miossec P, Vitse O, Zhou T, Schoser B, NEO-EXT investigators.
    Neurology; 2022 Aug 01; 99(5):e536-e548. PubMed ID: 35618441
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