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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.
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. 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 cipaglucosidase alfa plus miglustat versus alglucosidase alfa plus placebo in late-onset Pompe disease (PROPEL): an international, randomised, double-blind, parallel-group, phase 3 trial.
Schoser B, Roberts M, Byrne BJ, Sitaraman S, Jiang H, Laforêt P, Toscano A, Castelli J, Díaz-Manera J, Goldman M, van der Ploeg AT, Bratkovic D, Kuchipudi S, Mozaffar T, Kishnani PS; PROPEL Study Group. Schoser B, et al. Lancet Neurol. 2021 Dec;20(12):1027-1037. doi: 10.1016/S1474-4422(21)00331-8. Lancet Neurol. 2021. PMID: 34800400 Clinical Trial.
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. 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.
Membrane Proteome-Wide Screening of Autoantibodies in CIDP Using Human Cell Microarray Technology.
Caballero-Ávila M, Lleixà C, Pascual-Goñi E, Martín-Aguilar L, Vidal-Fernandez N, Tejada-Illa C, Collet-Vidiella R, Rojas-Garcia R, Cortés-Vicente E, Turon-Sans J, Gallardo E, Olivé M, Vesperinas A, Carbayo Á, Llansó L, Martinez-Martinez L, Shock A, Christodoulou L, Dizier B, Freeth J, Soden J, Dawson S, Querol L. Caballero-Ávila M, et al. Among authors: vidal fernandez n. Neurol Neuroimmunol Neuroinflamm. 2024 May;11(3):e200216. doi: 10.1212/NXI.0000000000200216. Epub 2024 Mar 14. Neurol Neuroimmunol Neuroinflamm. 2024. PMID: 38484217 Free PMC article.
mRNA COVID-19 Vaccination Does Not Exacerbate Symptoms or Trigger Neural Antibody Responses in Multiple Sclerosis.
Blanco Y, Escudero D, Lleixà C, Llufriu S, Egri N, García RR, Alba M, Aguilar E, Artola M, Aldea Novo M, Alvarez S, Caballero E, Cabrera-Maqueda JM, Fonseca E, Guasp M, Hernando A, Martinez-Hernandez E, Olivé-Cirera G, Lopez-Contreras J, Martín-Aguilar L, Martinez-Martinez L, Rombauts A, Rodés M, Sabater L, Sepulveda M, Solana E, Tejada-Illa C, Vidal-Fernández N, Vilella A, Fortuny C, Armangué T, Dalmau JO, Querol L, Saiz A. Blanco Y, et al. Among authors: vidal fernandez n. Neurol Neuroimmunol Neuroinflamm. 2023 Sep 7;10(6):e200163. doi: 10.1212/NXI.0000000000200163. Print 2023 Nov. Neurol Neuroimmunol Neuroinflamm. 2023. PMID: 37679040 Free PMC 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. 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.