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2021 1
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Safety and clinical activity of autologous RNA chimeric antigen receptor T-cell therapy in myasthenia gravis (MG-001): a prospective, multicentre, open-label, non-randomised phase 1b/2a study.
Granit V, Benatar M, Kurtoglu M, Miljković MD, Chahin N, Sahagian G, Feinberg MH, Slansky A, Vu T, Jewell CM, Singer MS, Kalayoglu MV, Howard JF Jr, Mozaffar T; MG-001 Study Team. Granit V, et al. Lancet Neurol. 2023 Jul;22(7):578-590. doi: 10.1016/S1474-4422(23)00194-1. Lancet Neurol. 2023. PMID: 37353278 Free PMC article. Clinical Trial.
Inclusion body myositis: evolving concepts.
Perez-Rosendahl M, Mozaffar T. Perez-Rosendahl M, et al. Curr Opin Neurol. 2022 Oct 1;35(5):604-610. doi: 10.1097/WCO.0000000000001095. Curr Opin Neurol. 2022. PMID: 36069417 Free PMC article. Review.
Editorial: Inflammatory muscle diseases: an update.
Tanboon J, Needham M, Mozaffar T, Stenzel W, Nishino I. Tanboon J, et al. Front Neurol. 2023 Aug 8;14:1259275. doi: 10.3389/fneur.2023.1259275. eCollection 2023. Front Neurol. 2023. PMID: 37614973 Free PMC article. No abstract available.
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. 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.
GNE myopathy: History, etiology, and treatment trials.
Mullen J, Alrasheed K, Mozaffar T. Mullen J, et al. Front Neurol. 2022 Oct 18;13:1002310. doi: 10.3389/fneur.2022.1002310. eCollection 2022. Front Neurol. 2022. PMID: 36330422 Free PMC article. Review.
Safety and Efficacy of Topiramate in Individuals With Cryptogenic Sensory Peripheral Neuropathy With Metabolic Syndrome: The TopCSPN Randomized Clinical Trial.
Smith AG, Singleton JR, Aperghis A, Coffey CS, Creigh P, Cudkowicz M, Conwit R, Ecklund D, Fedler JK, Gudjonsdottir A, Hauer P, Herrmann DN, Kearney M, Kissel J, Klingner E, Quick A, Revere C, Stino A; NeuroNEXT NN108 TopCSPN Study Team. Smith AG, et al. JAMA Neurol. 2023 Dec 1;80(12):1334-1343. doi: 10.1001/jamaneurol.2023.3711. JAMA Neurol. 2023. PMID: 37870862 Free PMC article. Clinical Trial.
Pompe Disease: a Clinical, Diagnostic, and Therapeutic Overview.
Stevens D, Milani-Nejad S, Mozaffar T. Stevens D, et al. Curr Treat Options Neurol. 2022 Nov;24(11):573-588. doi: 10.1007/s11940-022-00736-1. Epub 2022 Aug 4. Curr Treat Options Neurol. 2022. PMID: 36969713 Free PMC article.
Immunophenotyping of Inclusion Body Myositis Blood T and NK Cells.
Goyal NA, Coulis G, Duarte J, Farahat PK, Mannaa AH, Cauchii J, Irani T, Araujo N, Wang L, Wencel M, Li V, Zhang L, Greenberg SA, Mozaffar T, Villalta SA. Goyal NA, et al. Neurology. 2022 Mar 29;98(13):e1374-e1383. doi: 10.1212/WNL.0000000000200013. Epub 2022 Feb 7. Neurology. 2022. PMID: 35131904 Free PMC article.
Provisional practice recommendation for the management of myopathy in VCP-associated multisystem proteinopathy.
Roy B, Peck A, Evangelista T, Pfeffer G, Wang L, Diaz-Manera J, Korb M, Wicklund MP, Milone M, Freimer M, Kushlaf H, Villar-Quiles RN, Stojkovic T, Needham M, Palmio J, Lloyd TE, Keung B, Mozaffar T, Weihl CC, Kimonis V. Roy B, et al. Ann Clin Transl Neurol. 2023 May;10(5):686-695. doi: 10.1002/acn3.51760. Epub 2023 Apr 7. Ann Clin Transl Neurol. 2023. PMID: 37026610 Free PMC article. Review.
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