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Journal Abstract Search

179 related items for PubMed ID: 36214004

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  • 4. 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; 20(12):1012-1026. PubMed ID: 34800399
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  • 8. 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; 271(8):5433-5446. PubMed ID: 38896264
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  • 12. Prospective exploratory muscle biopsy, imaging, and functional assessment in patients with late-onset Pompe disease treated with alglucosidase alfa: The EMBASSY Study.
    van der Ploeg A, Carlier PG, Carlier RY, Kissel JT, Schoser B, Wenninger S, Pestronk A, Barohn RJ, Dimachkie MM, Goker-Alpan O, Mozaffar T, Pena LD, Simmons Z, Straub V, Guglieri M, Young P, Boentert M, Baudin PY, Wens S, Shafi R, Bjartmar C, Thurberg BL.
    Mol Genet Metab; 2016 Sep; 119(1-2):115-23. PubMed ID: 27473031
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  • 13. 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; 264(4):621-630. PubMed ID: 27372449
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  • 16. 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; 29(3):167-186. PubMed ID: 30770310
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  • 18. Pharmacokinetics of Alglucosidase Alfa Manufactured at the 4000-L Scale in Participants with Pompe Disease: A Phase 3/4 Open-Label Study.
    Nicolas X, Hurbin F, Periquet M, Richards S, Sensinger C, Welch K, An Haack K.
    Clin Pharmacol Drug Dev; 2023 Dec; 12(12):1185-1193. PubMed ID: 37705424
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  • 20. Higher dose alglucosidase alfa is associated with improved overall survival in infantile-onset Pompe disease (IOPD): data from the Pompe Registry.
    Kishnani PS, Kronn D, Suwazono S, Broomfield A, Llerena J, Al-Hassnan ZN, Batista JL, Wilson KM, Periquet M, Daba N, Hahn A, Chien YH.
    Orphanet J Rare Dis; 2023 Dec 06; 18(1):381. PubMed ID: 38057861
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