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Journal Abstract Search

295 related items for PubMed ID: 34800400

  • 1. 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
    [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
    [Abstract] [Full Text] [Related]

  • 4. 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
    [Abstract] [Full Text] [Related]

  • 5. Enzyme replacement therapy for infantile-onset Pompe disease.
    Chen M, Zhang L, Quan S.
    Cochrane Database Syst Rev; 2017 Nov 20; 11(11):CD011539. PubMed ID: 29155436
    [Abstract] [Full Text] [Related]

  • 6. 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 20; 13(10):e240045. PubMed ID: 39287071
    [Abstract] [Full Text] [Related]

  • 7. 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]

  • 8. Improved Enzyme Replacement Therapy with Cipaglucosidase Alfa/Miglustat in Infantile Pompe Disease.
    Fiege L, Duran I, Marquardt T.
    Pharmaceuticals (Basel); 2023 Aug 23; 16(9):. PubMed ID: 37765007
    [Abstract] [Full Text] [Related]

  • 9. Cipaglucosidase Alfa: First Approval.
    Blair HA.
    Drugs; 2023 Jun 23; 83(8):739-745. PubMed ID: 37184753
    [Abstract] [Full Text] [Related]

  • 10. Safety, tolerability, pharmacokinetics, pharmacodynamics, and exploratory efficacy of the novel enzyme replacement therapy avalglucosidase alfa (neoGAA) in treatment-naïve and alglucosidase alfa-treated patients with late-onset Pompe disease: A phase 1, open-label, multicenter, multinational, ascending dose study.
    Pena LDM, Barohn RJ, Byrne BJ, Desnuelle C, Goker-Alpan O, Ladha S, Laforêt P, Mengel KE, Pestronk A, Pouget J, Schoser B, Straub V, Trivedi J, Van Damme P, Vissing J, Young P, Kacena K, Shafi R, Thurberg BL, Culm-Merdek K, van der Ploeg AT, NEO1 Investigator Group.
    Neuromuscul Disord; 2019 Mar 23; 29(3):167-186. PubMed ID: 30770310
    [Abstract] [Full Text] [Related]

  • 11. Long-term safety and efficacy of cipaglucosidase alfa plus miglustat in individuals living with Pompe disease: an open-label phase I/II study (ATB200-02).
    Byrne BJ, Schoser B, Kishnani PS, Bratkovic D, Clemens PR, Goker-Alpan O, Ming X, Roberts M, Vorgerd M, Sivakumar K, van der Ploeg AT, Goldman M, Wright J, Holdbrook F, Jain V, Benjamin ER, Johnson F, Das SS, Wasfi Y, Mozaffar T.
    J Neurol; 2024 Apr 23; 271(4):1787-1801. PubMed ID: 38057636
    [Abstract] [Full Text] [Related]

  • 12. 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 23; 387(2):188-203. PubMed ID: 37679046
    [Abstract] [Full Text] [Related]

  • 13. 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 Nov 23; 11(2):80-85. PubMed ID: 39318718
    [Abstract] [Full Text] [Related]

  • 14. 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]

  • 15. 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]

  • 16. Open-label extension study following the Late-Onset Treatment Study (LOTS) of alglucosidase alfa.
    van der Ploeg AT, Barohn R, Carlson L, Charrow J, Clemens PR, Hopkin RJ, Kishnani PS, Laforêt P, Morgan C, Nations S, Pestronk A, Plotkin H, Rosenbloom BE, Sims KB, Tsao E.
    Mol Genet Metab; 2012 Nov 24; 107(3):456-61. PubMed ID: 23031366
    [Abstract] [Full Text] [Related]

  • 17. 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 24; 25(2):100328. PubMed ID: 36542086
    [Abstract] [Full Text] [Related]

  • 18. 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 24; 141(2):108121. PubMed ID: 38184428
    [Abstract] [Full Text] [Related]

  • 19. 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]

  • 20. 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 15; 257(1):91-7. PubMed ID: 19649685
    [Abstract] [Full Text] [Related]

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