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Journal Abstract Search

188 related items for PubMed ID: 38313679

  • 1. Real-world outcomes from a series of patients with late onset Pompe disease who switched from alglucosidase alfa to avalglucosidase alfa.
    Carter C, Boggs T, Case LE, Kishnani P.
    Front Genet; 2024; 15():1309146. PubMed ID: 38313679
    [Abstract] [Full Text] [Related]

  • 2. 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
    [Abstract] [Full Text] [Related]

  • 3. Safety and efficacy of avalglucosidase alfa versus alglucosidase alfa in patients with late-onset Pompe disease (COMET): a phase 3, randomised, multicentre trial.
    Diaz-Manera J, Kishnani PS, Kushlaf H, Ladha S, Mozaffar T, Straub V, Toscano A, van der Ploeg AT, Berger KI, Clemens PR, Chien YH, Day JW, Illarioshkin S, Roberts M, Attarian S, Borges JL, Bouhour F, Choi YC, Erdem-Ozdamar S, Goker-Alpan O, Kostera-Pruszczyk A, Haack KA, Hug C, Huynh-Ba O, Johnson J, Thibault N, Zhou T, Dimachkie MM, Schoser B, COMET Investigator Group.
    Lancet Neurol; 2021 Dec 12; 20(12):1012-1026. PubMed ID: 34800399
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  • 5. 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
    [Abstract] [Full Text] [Related]

  • 6. 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 01; 141(2):108121. PubMed ID: 38184428
    [Abstract] [Full Text] [Related]

  • 7. Efficacy and Safety of Avalglucosidase Alfa in Patients With Late-Onset Pompe Disease After 97 Weeks: A Phase 3 Randomized Clinical Trial.
    Kishnani PS, Diaz-Manera J, Toscano A, Clemens PR, Ladha S, Berger KI, Kushlaf H, Straub V, Carvalho G, Mozaffar T, Roberts M, Attarian S, Chien YH, Choi YC, Day JW, Erdem-Ozdamar S, Illarioshkin S, Goker-Alpan O, Kostera-Pruszczyk A, van der Ploeg AT, An Haack K, Huynh-Ba O, Tammireddy S, Thibault N, Zhou T, Dimachkie MM, Schoser B, COMET Investigator Group.
    JAMA Neurol; 2023 Jun 01; 80(6):558-567. PubMed ID: 37036722
    [Abstract] [Full Text] [Related]

  • 8. 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 01; 31(7):e16292. PubMed ID: 38587143
    [Abstract] [Full Text] [Related]

  • 9. Safety and efficacy of avalglucosidase alfa in individuals with infantile-onset Pompe disease enrolled in the phase 2, open-label Mini-COMET study: The 6-month primary analysis report.
    Kishnani PS, Kronn D, Brassier A, Broomfield A, Davison J, Hahn SH, Kumada S, Labarthe F, Ohki H, Pichard S, Prakalapakorn SG, Haack KA, Kittner B, Meng X, Sparks S, Wilson C, Zaher A, Chien YH, Mini-COMET Investigators.
    Genet Med; 2023 Feb 01; 25(2):100328. PubMed ID: 36542086
    [Abstract] [Full Text] [Related]

  • 10. Clinical insight meets scientific innovation to develop a next generation ERT for Pompe disease.
    Kishnani PS, Chien YH, Berger KI, Thibault N, Sparks S.
    Mol Genet Metab; 2024 Feb 01; 143(1-2):108559. PubMed ID: 39154400
    [Abstract] [Full Text] [Related]

  • 11. 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 01; 264(4):621-630. PubMed ID: 27372449
    [Abstract] [Full Text] [Related]

  • 12. Transcriptomic characterization of clinical skeletal muscle biopsy from late-onset Pompe patients.
    Kinton S, Dufault MR, Zhang M, George K.
    Mol Genet Metab; 2023 Mar 01; 138(3):107526. PubMed ID: 36774918
    [Abstract] [Full Text] [Related]

  • 13. 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 01; 20(12):1027-1037. PubMed ID: 34800400
    [Abstract] [Full Text] [Related]

  • 14. 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 01; 271(5):2810-2823. PubMed ID: 38418563
    [Abstract] [Full Text] [Related]

  • 15. Increasing Enzyme Mannose-6-Phosphate Levels but Not Miglustat Coadministration Enhances the Efficacy of Enzyme Replacement Therapy in Pompe Mice.
    Anding A, Kinton S, Baranowski K, Brezzani A, De Busser H, Dufault MR, Finn P, Keefe K, Tetrault T, Li Y, Qiu W, Raes K, Vitse O, Zhang M, Ziegler R, Sardi SP, Hunter B, George K.
    J Pharmacol Exp Ther; 2023 Nov 01; 387(2):188-203. PubMed ID: 37679046
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  • 16. Enzyme replacement therapy in late-onset Pompe disease: a systematic literature review.
    Toscano A, Schoser B.
    J Neurol; 2013 Apr 01; 260(4):951-9. PubMed ID: 22926164
    [Abstract] [Full Text] [Related]

  • 17. Post-hoc Nonparametric Analysis of Forced Vital Capacity in the COMET Trial Demonstrates Superiority of Avalglucosidase Alfa vs Alglucosidase Alfa.
    Boentert M, Campana ES, Attarian S, Diaz-Manera J, Dimachkie MM, Periquet M, Thibault N, Miossec P, Zhou T, Berger KI.
    J Neuromuscul Dis; 2024 Apr 01; 11(2):369-374. PubMed ID: 38160363
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  • 20. Pompe disease: Unmet needs and emerging therapies.
    George KA, Anding AL, van der Flier A, Tomassy GS, Berger KI, Zhang TY, Sardi SP.
    Mol Genet Metab; 2024 Nov 01; 143(3):108590. PubMed ID: 39418752
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