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Long-term safety and efficacy of cipaglucosidase alfa plus miglustat in individuals living with Pompe disease: an open-label phase I/II study (ATB200-02).
Byrne BJ, Schoser B, Kishnani PS, Bratkovic D, Clemens PR, Goker-Alpan O, Ming X, Roberts M, Vorgerd M, Sivakumar K, van der Ploeg AT, Goldman M, Wright J, Holdbrook F, Jain V, Benjamin ER, Johnson F, Das SS, Wasfi Y, Mozaffar T. Byrne BJ, et al. Among authors: schoser b. J Neurol. 2024 Apr;271(4):1787-1801. doi: 10.1007/s00415-023-12096-0. Epub 2023 Dec 6. J Neurol. 2024. PMID: 38057636 Free PMC article. Clinical Trial.
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.
Toscano A, Pollissard L, Msihid J, van der Beek N, Kishnani PS, Dimachkie MM, Berger KI, DasMahapatra P, Thibault N, Hamed A, Zhou T, Haack KA, Schoser B. Toscano A, et al. Among authors: schoser b. 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.
Cipaglucosidase alfa plus miglustat: linking mechanism of action to clinical outcomes in late-onset Pompe disease.
Byrne BJ, Parenti G, Schoser B, van der Ploeg AT, Do H, Fox B, Goldman M, Johnson FK, Kang J, Mehta N, Mondick J, Sheikh MO, Sitaraman Das S, Tuske S, Brudvig J, Weimer JM, Mozaffar T. Byrne BJ, et al. Among authors: schoser b. Front Neurol. 2024 Oct 18;15:1451512. doi: 10.3389/fneur.2024.1451512. eCollection 2024. Front Neurol. 2024. PMID: 39494167 Free PMC article.
Cognitive behavioural therapy with optional graded exercise therapy in patients with severe fatigue with myotonic dystrophy type 1: a multicentre, single-blind, randomised trial.
Okkersen K, Jimenez-Moreno C, Wenninger S, Daidj F, Glennon J, Cumming S, Littleford R, Monckton DG, Lochmüller H, Catt M, Faber CG, Hapca A, Donnan PT, Gorman G, Bassez G, Schoser B, Knoop H, Treweek S, van Engelen BGM; OPTIMISTIC consortium. Okkersen K, et al. Among authors: schoser b. Lancet Neurol. 2018 Aug;17(8):671-680. doi: 10.1016/S1474-4422(18)30203-5. Epub 2018 Jun 19. Lancet Neurol. 2018. PMID: 29934199 Free article. Clinical Trial.
[No title available]
[No authors listed] [No authors listed] PMID: 39468915
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: schoser b. 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.
Effects of gene replacement therapy with resamirigene bilparvovec (AT132) on skeletal muscle pathology in X-linked myotubular myopathy: results from a substudy of the ASPIRO open-label clinical trial.
Lawlor MW, Schoser B, Margeta M, Sewry CA, Jones KA, Shieh PB, Kuntz NL, Smith BK, Dowling JJ, Müller-Felber W, Bönnemann CG, Seferian AM, Blaschek A, Neuhaus S, Foley AR, Saade DN, Tsuchiya E, Qasim UR, Beatka M, Prom MJ, Ott E, Danielson S, Krakau P, Kumar SN, Meng H, Vanden Avond M, Wells C, Gordish-Dressman H, Beggs AH, Christensen S, Conner E, James ES, Lee J, Sadhu C, Miller W, Sepulveda B, Varfaj F, Prasad S, Rico S. Lawlor MW, et al. Among authors: schoser b. EBioMedicine. 2024 Jan;99:104894. doi: 10.1016/j.ebiom.2023.104894. Epub 2023 Dec 12. EBioMedicine. 2024. PMID: 38086156 Free PMC article.
361 results