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172 results

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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. Schoser B, et al. Among authors: claeys kg. J Neurol. 2024 May;271(5):2810-2823. doi: 10.1007/s00415-024-12236-0. Epub 2024 Feb 28. J Neurol. 2024. PMID: 38418563 Free PMC article. Clinical Trial.
Randomized phase 2 study of FcRn antagonist efgartigimod in generalized myasthenia gravis.
Howard JF Jr, Bril V, Burns TM, Mantegazza R, Bilinska M, Szczudlik A, Beydoun S, Garrido FJRR, Piehl F, Rottoli M, Van Damme P, Vu T, Evoli A, Freimer M, Mozaffar T, Ward ES, Dreier T, Ulrichts P, Verschueren K, Guglietta A, de Haard H, Leupin N, Verschuuren JJGM; Efgartigimod MG Study Group. Howard JF Jr, et al. Neurology. 2019 Jun 4;92(23):e2661-e2673. doi: 10.1212/WNL.0000000000007600. Epub 2019 May 22. Neurology. 2019. PMID: 31118245 Free PMC article. Clinical Trial.
Improving outcome measures in late onset Pompe disease: Modified Rasch-Built Pompe-Specific Activity scale.
van Kooten HA, Horton MC, Wenninger S, Babačić H, Schoser B, Lefeuvre C, Taouagh N, Laforêt P, Segovia S, Díaz-Manera J, Claeys KG, Mongini T, Musumeci O, Toscano A, Hundsberger T, Brusse E, van Doorn PA, van der Ploeg AT, van der Beek NAME; European Pompe Consortium study group on outcome measures. van Kooten HA, et al. Among authors: claeys kg. Eur J Neurol. 2024 Dec;31(12):e16397. doi: 10.1111/ene.16397. Epub 2024 Aug 28. Eur J Neurol. 2024. PMID: 39205420 Free PMC article.
Original research: Second IVIg course in Guillain-Barré syndrome with poor prognosis: the non-randomised ISID study.
Verboon C, van den Berg B, Cornblath DR, Venema E, Gorson KC, Lunn MP, Lingsma H, Van den Bergh P, Harbo T, Bateman K, Pereon Y, Sindrup SH, Kusunoki S, Miller J, Islam Z, Hartung HP, Chavada G, Jacobs BC, Hughes RAC, van Doorn PA; IGOS Consortium. Verboon C, et al. J Neurol Neurosurg Psychiatry. 2020 Feb;91(2):113-121. doi: 10.1136/jnnp-2019-321496. Epub 2019 Oct 5. J Neurol Neurosurg Psychiatry. 2020. PMID: 31586949
Efficacy, safety and tolerability of rozanolixizumab in patients with chronic inflammatory demyelinating polyradiculoneuropathy: a randomised, subject-blind, investigator-blind, placebo-controlled, phase 2a trial and open-label extension study.
Querol L, De Sèze J, Dysgaard T, Levine T, Rao TH, Rivner M, Hartung HP, Kiessling P, Shimizu S, Marmol D, Bozorg A, Colson AO, Massow U, Eftimov F; CIDP01 Study Investigators. Querol L, et al. J Neurol Neurosurg Psychiatry. 2024 Aug 16;95(9):845-854. doi: 10.1136/jnnp-2023-333112. J Neurol Neurosurg Psychiatry. 2024. PMID: 38729747 Free PMC article. Clinical Trial.
Regional variation of Guillain-Barré syndrome.
Doets AY, Verboon C, van den Berg B, Harbo T, Cornblath DR, Willison HJ, Islam Z, Attarian S, Barroso FA, Bateman K, Benedetti L, van den Bergh P, Casasnovas C, Cavaletti G, Chavada G, Claeys KG, Dardiotis E, Davidson A, van Doorn PA, Feasby TE, Galassi G, Gorson KC, Hartung HP, Hsieh ST, Hughes RAC, Illa I, Islam B, Kusunoki S, Kuwabara S, Lehmann HC, Miller JAL, Mohammad QD, Monges S, Nobile Orazio E, Pardo J, Pereon Y, Rinaldi S, Querol L, Reddel SW, Reisin RC, Shahrizaila N, Sindrup SH, Waqar W, Jacobs BC; IGOS Consortium. Doets AY, et al. Among authors: claeys kg. Brain. 2018 Oct 1;141(10):2866-2877. doi: 10.1093/brain/awy232. Brain. 2018. PMID: 30247567 Free article.
Current treatment practice of Guillain-Barré syndrome.
Verboon C, Doets AY, Galassi G, Davidson A, Waheed W, Péréon Y, Shahrizaila N, Kusunoki S, Lehmann HC, Harbo T, Monges S, Van den Bergh P, Willison HJ, Cornblath DR, Jacobs BC; IGOS Consortium. Verboon C, et al. Neurology. 2019 Jul 2;93(1):e59-e76. doi: 10.1212/WNL.0000000000007719. Epub 2019 Jun 7. Neurology. 2019. PMID: 31175208 Free article.
Electrodiagnostic subtyping in Guillain-Barré syndrome patients in the International Guillain-Barré Outcome Study.
Arends S, Drenthen J, de Koning L, van den Bergh P, Hadden RDM, Kuwabara S, Reisin RC, Shahrizaila N, Ajroud-Driss S, Antonini G, Attarian S, Balducci C, Bertorini T, Brannagan TH, Cavaletti G, Chao CC, Chavada G, Dillmann KU, Dimachkie MM, Galassi G, Gutiérrez-Gutiérrez G, Harbo T, Islam B, Islam Z, Katzberg H, Kusunoki S, Manganelli F, Miller JAL, Pardo J, Pereon Y, Rajabally YA, Sindrup S, Stettner M, Uncini A, Verhamme C, Vytopil M, Waheed W, Jacobs BC, Cornblath DR; IGOS Consortium. Arends S, et al. Eur J Neurol. 2024 Sep;31(9):e16335. doi: 10.1111/ene.16335. Epub 2024 Jul 4. Eur J Neurol. 2024. PMID: 38965709 Free PMC article.
International Guillain-Barré Syndrome Outcome Study: protocol of a prospective observational cohort study on clinical and biological predictors of disease course and outcome in Guillain-Barré syndrome.
Jacobs BC, van den Berg B, Verboon C, Chavada G, Cornblath DR, Gorson KC, Harbo T, Hartung HP, Hughes RAC, Kusunoki S, van Doorn PA, Willison HJ; IGOS Consortium. Jacobs BC, et al. J Peripher Nerv Syst. 2017 Jun;22(2):68-76. doi: 10.1111/jns.12209. J Peripher Nerv Syst. 2017. PMID: 28406555 Free article.
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. Kishnani PS, et al. Among authors: claeys kg. J Patient Rep Outcomes. 2024 Nov 13;8(1):132. doi: 10.1186/s41687-024-00805-w. J Patient Rep Outcomes. 2024. PMID: 39535661 Free PMC article. Clinical Trial.
172 results