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126 results

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Safety and efficacy of zilucoplan in patients with generalised myasthenia gravis (RAISE): a randomised, double-blind, placebo-controlled, phase 3 study.
Howard JF Jr, Bresch S, Genge A, Hewamadduma C, Hinton J, Hussain Y, Juntas-Morales R, Kaminski HJ, Maniaol A, Mantegazza R, Masuda M, Sivakumar K, Śmiłowski M, Utsugisawa K, Vu T, Weiss MD, Zajda M, Boroojerdi B, Brock M, de la Borderie G, Duda PW, Lowcock R, Vanderkelen M, Leite MI; RAISE Study Team. Howard JF Jr, et al. Among authors: genge a. Lancet Neurol. 2023 May;22(5):395-406. doi: 10.1016/S1474-4422(23)00080-7. Lancet Neurol. 2023. PMID: 37059508 Clinical Trial.
Safety and efficacy of lithium in combination with riluzole for treatment of amyotrophic lateral sclerosis: a randomised, double-blind, placebo-controlled trial.
Aggarwal SP, Zinman L, Simpson E, McKinley J, Jackson KE, Pinto H, Kaufman P, Conwit RA, Schoenfeld D, Shefner J, Cudkowicz M; Northeast and Canadian Amyotrophic Lateral Sclerosis consortia. Aggarwal SP, et al. Lancet Neurol. 2010 May;9(5):481-8. doi: 10.1016/S1474-4422(10)70068-5. Epub 2010 Apr 1. Lancet Neurol. 2010. PMID: 20363190 Free PMC article. Clinical Trial.
Investigating Late-Onset Pompe Prevalence in Neuromuscular Medicine Academic Practices: The IPaNeMA Study.
Wencel M, Shaibani A, Goyal NA, Dimachkie MM, Trivedi J, Johnson NE, Gutmann L, Wicklund MP, Bandyopadhay S, Genge AL, Freimer ML, Goyal N, Pestronk A, Florence J, Karam C, Ralph JW, Rasheed Z, Hays M, Hopkins S, Mozaffar T. Wencel M, et al. Among authors: genge al. Neurol Genet. 2021 Oct 18;7(6):e623. doi: 10.1212/NXG.0000000000000623. eCollection 2021 Dec. Neurol Genet. 2021. PMID: 36299500 Free PMC article.
Efficacy and Safety of Avalglucosidase Alfa in Patients With Late-Onset Pompe Disease After 97 Weeks: A Phase 3 Randomized Clinical Trial.
Kishnani PS, Diaz-Manera J, Toscano A, Clemens PR, Ladha S, Berger KI, Kushlaf H, Straub V, Carvalho G, Mozaffar T, Roberts M, Attarian S, Chien YH, Choi YC, Day JW, Erdem-Ozdamar S, Illarioshkin S, Goker-Alpan O, Kostera-Pruszczyk A, van der Ploeg AT, An Haack K, Huynh-Ba O, Tammireddy S, Thibault N, Zhou T, Dimachkie MM, Schoser B; COMET Investigator Group. Kishnani PS, et al. JAMA Neurol. 2023 Jun 1;80(6):558-567. doi: 10.1001/jamaneurol.2023.0552. JAMA Neurol. 2023. PMID: 37036722 Free PMC article. Clinical Trial.
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. Lancet Neurol. 2023 May;22(5):383-394. doi: 10.1016/S1474-4422(23)00077-7. Lancet Neurol. 2023. PMID: 37059507 Clinical Trial.
Efficacy and safety of iscalimab, a novel anti-CD40 monoclonal antibody, in moderate-to-severe myasthenia gravis: A phase 2 randomized study.
GomezMancilla B, Meriggioli MN, Genge A, Roubenoff R, Espié P, Dupuy C, Hartmann N, Pezous N, Kinhikar A, Tichy M, Dionne A, Vissing J, Andersen H, Schoser B, Meisel A, Jordan B, Devlikamova F, Poverennova I, Stuchevskaya F, Lin TS, Rush JS, Gergely P. GomezMancilla B, et al. Among authors: genge a. J Clin Neurosci. 2024 Jan;119:76-84. doi: 10.1016/j.jocn.2023.11.013. Epub 2023 Nov 20. J Clin Neurosci. 2024. PMID: 37988976 Clinical Trial.
Long-term safety and efficacy of zilucoplan in patients with generalized myasthenia gravis: interim analysis of the RAISE-XT open-label extension study.
Howard JF Jr, Bresch S, Farmakidis C, Freimer M, Genge A, Hewamadduma C, Hinton J, Hussain Y, Juntas-Morales R, Kaminski HJ, Maniaol A, Mantegazza R, Masuda M, Nowak RJ, Sivakumar K, Śmiłowski M, Utsugisawa K, Vu T, Weiss MD, Zajda M, Bloemers J, Boroojerdi B, Brock M, de la Borderie G, Duda PW, Vanderkelen M, Leite MI; RAISE-XT Study Team*. Howard JF Jr, et al. Among authors: genge a. Ther Adv Neurol Disord. 2024 Apr 17;17:17562864241243186. doi: 10.1177/17562864241243186. eCollection 2024. Ther Adv Neurol Disord. 2024. PMID: 38638673 Free PMC article.
126 results