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Characteristics of Patients With Late-Onset Pompe Disease in France: Insights From the French Pompe Registry in 2022.
Lefeuvre C, De Antonio M, Bouhour F, Tard C, Salort-Campana E, Lagrange E, Behin A, Sole G, Noury JB, Sacconi S, Magot A, Nadaj-Pakleza A, Lacour A, Beltran S, Spinazzi M, Cintas P, Renard D, Michaud M, Bedat-Millet AL, Prigent H, Taouagh N, Arrassi A, Hamroun D, Attarian S, Laforêt P; for Pompe Study Group. Lefeuvre C, et al. Among authors: sacconi s. Neurology. 2023 Aug 29;101(9):e966-e977. doi: 10.1212/WNL.0000000000207547. Epub 2023 Jul 7. Neurology. 2023. PMID: 37419682
Long-term exposure to Myozyme results in a decrease of anti-drug antibodies in late-onset Pompe disease patients.
Masat E, Laforêt P, De Antonio M, Corre G, Perniconi B, Taouagh N, Mariampillai K, Amelin D, Mauhin W, Hogrel JY, Caillaud C, Ronzitti G, Puzzo F, Kuranda K, Colella P, Mallone R, Benveniste O, Mingozzi F; French Pompe Registry Study Group. Masat E, et al. Sci Rep. 2016 Nov 4;6:36182. doi: 10.1038/srep36182. Sci Rep. 2016. PMID: 27812025 Free PMC article.
European consensus for starting and stopping enzyme replacement therapy in adult patients with Pompe disease: a 10-year experience.
van der Ploeg AT, Kruijshaar ME, Toscano A, Laforêt P, Angelini C, Lachmann RH, Pascual Pascual SI, Roberts M, Rösler K, Stulnig T, van Doorn PA, Van den Bergh PYK, Vissing J, Schoser B; European Pompe Consortium. van der Ploeg AT, et al. Eur J Neurol. 2017 Jun;24(6):768-e31. doi: 10.1111/ene.13285. Epub 2017 May 6. Eur J Neurol. 2017. PMID: 28477382 Review.
Effect of enzyme replacement therapy with alglucosidase alfa (Myozyme®) in 12 patients with advanced late-onset Pompe disease.
Papadopoulos C, Orlikowski D, Prigent H, Lacour A, Tard C, Furby A, Praline J, Solé G, Hogrel JY, De Antonio M, Semplicini C, Deibener-Kaminsky J, Kaminsky P, Eymard B, Taouagh N, Perniconi B, Hamroun D, Laforêt P; French Pompe Study Group. Papadopoulos C, et al. Mol Genet Metab. 2017 Sep;122(1-2):80-85. doi: 10.1016/j.ymgme.2017.06.007. Epub 2017 Jun 20. Mol Genet Metab. 2017. PMID: 28648663
Role of autoantibody levels as biomarkers in the management of patients with myasthenia gravis: A systematic review and expert appraisal.
Meisel A, Baggi F, Behin A, Evoli A, Kostera-Pruszczyk A, Mantegazza R, Morales RJ, Punga AR, Sacconi S, Schroeter M, Verschuuren J, Crathorne L, Holmes K, Leite MI. Meisel A, et al. Among authors: sacconi s. Eur J Neurol. 2023 Jan;30(1):266-282. doi: 10.1111/ene.15565. Epub 2022 Oct 7. Eur J Neurol. 2023. PMID: 36094738 Review.
Reply to the Letter to the Editor in response to "Role of autoantibody levels as biomarkers in the management of patients with myasthenia gravis: A systematic review and expert appraisal".
Meisel A, Baggi F, Behin A, Evoli A, Kostera-Pruszczyk A, Mantegazza R, Juntas Morales R, Punga AR, Sacconi S, Schroeter M, Verschuuren J, Crathorne L, Holmes K, Leite MI. Meisel A, et al. Among authors: sacconi s. Eur J Neurol. 2023 Apr;30(4):1162-1164. doi: 10.1111/ene.15690. Epub 2023 Feb 14. Eur J Neurol. 2023. PMID: 36692238 No abstract available.
Safety and efficacy of rozanolixizumab in patients with generalised myasthenia gravis (MycarinG): a randomised, double-blind, placebo-controlled, adaptive phase 3 study.
Bril V, Drużdż A, Grosskreutz J, Habib AA, Mantegazza R, Sacconi S, Utsugisawa K, Vissing J, Vu T, Boehnlein M, Bozorg A, Gayfieva M, Greve B, Woltering F, Kaminski HJ; MG0003 study team. Bril V, et al. Among authors: sacconi s. Lancet Neurol. 2023 May;22(5):383-394. doi: 10.1016/S1474-4422(23)00077-7. Lancet Neurol. 2023. PMID: 37059507 Clinical Trial.
Hypersensitivity infusion-associated reactions induced by enzyme replacement therapy in a cohort of patients with late-onset Pompe disease: An experience from the French Pompe Registry.
Lessard LER, Tard C, Salort-Campana E, Sacconi S, Béhin A, Bassez G, Orlikowski D, Merle P, Nollet S, Gallay L, Bérard F, Robinson P, Bouhour F, Laforêt P. Lessard LER, et al. Among authors: sacconi s. Mol Genet Metab. 2023 Jul;139(3):107611. doi: 10.1016/j.ymgme.2023.107611. Epub 2023 May 19. Mol Genet Metab. 2023. PMID: 37285781 Free article.
164 results