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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.
Mol Genet Metab. 2024 Feb;141(2):108121. doi: 10.1016/j.ymgme.2023.108121. Epub 2023 Dec 27.
Mol Genet Metab. 2024.
PMID: 38184428
Free article.
Clinical Trial.
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.
Diaz-Manera J, et al. Among authors: haack ka.
Lancet Neurol. 2021 Dec;20(12):1012-1026. doi: 10.1016/S1474-4422(21)00241-6.
Lancet Neurol. 2021.
PMID: 34800399
Clinical Trial.
Item in Clipboard
Forced vital capacity and cross-domain late-onset Pompe disease outcomes: an individual patient-level data meta-analysis.
Berger KI, Kanters S, Jansen JP, Stewart A, Sparks S, Haack KA, Bolzani A, Siliman G, Hamed A.
Berger KI, et al. Among authors: haack ka.
J Neurol. 2019 Sep;266(9):2312-2321. doi: 10.1007/s00415-019-09401-1. Epub 2019 Jun 11.
J Neurol. 2019.
PMID: 31187190
Free PMC article.
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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.
Dimachkie MM, et al. Among authors: haack ka.
Neurology. 2022 Aug 1;99(5):e536-e548. doi: 10.1212/WNL.0000000000200746.
Neurology. 2022.
PMID: 35618441
Free PMC article.
Item in Clipboard
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.
Kishnani PS, et al. Among authors: haack ka.
Genet Med. 2023 Feb;25(2):100328. doi: 10.1016/j.gim.2022.10.010. Epub 2022 Dec 21.
Genet Med. 2023.
PMID: 36542086
Free article.
Clinical Trial.
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Population pharmacokinetic modeling and dosing simulation of avalglucosidase alfa for selecting alternative dosing regimen in pediatric patients with late-onset pompe disease.
Tiraboschi G, Marchionni D, Tuffal G, Fabre D, Martinez JM, Haack KA, Miossec P, Kittner B, Daba N, Hurbin F.
Tiraboschi G, et al. Among authors: haack ka.
J Pharmacokinet Pharmacodyn. 2023 Dec;50(6):461-474. doi: 10.1007/s10928-023-09874-8. Epub 2023 Aug 3.
J Pharmacokinet Pharmacodyn. 2023.
PMID: 37535240
Free PMC article.
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