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Journal Abstract Search

169 related items for PubMed ID: 39287071

  • 1. 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; 13(10):e240045. PubMed ID: 39287071
    [Abstract] [Full Text] [Related]

  • 2. 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; 271(5):2810-2823. PubMed ID: 38418563
    [Abstract] [Full Text] [Related]

  • 3. Switching treatment to cipaglucosidase alfa plus miglustat positively affects patient-reported outcome measures in patients with late-onset Pompe disease.
    Kishnani PS, Byrne BJ, Claeys KG, Díaz-Manera J, Dimachkie MM, Kushlaf H, Mozaffar T, Roberts M, Schoser B, Hummel N, Kopiec A, Holdbrook F, Shohet S, Toscano A, PROPEL Study Group.
    J Patient Rep Outcomes; 2024 Nov 13; 8(1):132. PubMed ID: 39535661
    [Abstract] [Full Text] [Related]

  • 4. 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]

  • 5. 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 12; 20(12):1027-1037. PubMed ID: 34800400
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  • 7. 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 12; 387(2):188-203. PubMed ID: 37679046
    [Abstract] [Full Text] [Related]

  • 8. 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]

  • 9. 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
    [Abstract] [Full Text] [Related]

  • 10. 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 12; 31(7):e16292. PubMed ID: 38587143
    [Abstract] [Full Text] [Related]

  • 11. Cipaglucosidase Alfa: First Approval.
    Blair HA.
    Drugs; 2023 Jun 12; 83(8):739-745. PubMed ID: 37184753
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  • 12. 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 12; 143(3):108590. PubMed ID: 39418752
    [Abstract] [Full Text] [Related]

  • 13. Regulatory news: Cipaglucosidase alfa-atga (Pombiliti) coadministered with Miglustat (Opfolda) for adults with late-onset Pompe disease.
    Luquetti DV, Jeng LJB, Donohue KM, Maynard JW, Review TeamOffice of Biostatistics, Center for Drug Evaluation and Research, U.S. Food and Drug Administration, Silver Spring, Maryland, USA.Office of Clinical Pharmacology, Center for Drug Evaluation and Research, U.S. Food and Drug Administration, Silver Spring, Maryland, USA.Office of Pharmaceutical Quality, Center for Drug Evaluation and Research, U.S. Food and Drug Administration, Silver Spring, Maryland, USA..
    J Inherit Metab Dis; 2024 Jul 12; 47(4):578-581. PubMed ID: 38768612
    [No Abstract] [Full Text] [Related]

  • 14. 36 months observational clinical study of 38 adult Pompe disease patients under alglucosidase alfa enzyme replacement therapy.
    Regnery C, Kornblum C, Hanisch F, Vielhaber S, Strigl-Pill N, Grunert B, Müller-Felber W, Glocker FX, Spranger M, Deschauer M, Mengel E, Schoser B.
    J Inherit Metab Dis; 2012 Sep 12; 35(5):837-45. PubMed ID: 22290025
    [Abstract] [Full Text] [Related]

  • 15. 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 12; 257(1):91-7. PubMed ID: 19649685
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  • 17. Changes in forced vital capacity over ≤ 13 years among patients with late-onset Pompe disease treated with alglucosidase alfa: new modeling of real-world data from the Pompe Registry.
    Berger KI, Chien YH, Dubrovsky A, Kishnani PS, Llerena JC, Neilan E, Roberts M, Sheng B, Batista JL, Periquet M, Wilson KM, van der Ploeg AT.
    J Neurol; 2024 Aug 12; 271(8):5433-5446. PubMed ID: 38896264
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  • 20. Large variation in effects during 10 years of enzyme therapy in adults with Pompe disease.
    Harlaar L, Hogrel JY, Perniconi B, Kruijshaar ME, Rizopoulos D, Taouagh N, Canal A, Brusse E, van Doorn PA, van der Ploeg AT, Laforêt P, van der Beek NAME.
    Neurology; 2019 Nov 05; 93(19):e1756-e1767. PubMed ID: 31619483
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