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211 results

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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. Among authors: mozaffar t. Lancet Neurol. 2023 Jul;22(7):578-590. doi: 10.1016/S1474-4422(23)00194-1. Lancet Neurol. 2023. PMID: 37353278 Clinical Trial.
The Miami Framework for ALS and related neurodegenerative disorders: an integrated view of phenotype and biology.
Benatar M, Wuu J, Huey ED, McMillan CT, Petersen RC, Postuma R, McHutchison C, Dratch L, Arias JJ, Crawley A, Houlden H, McDermott MP, Cai X, Thakur N, Boxer A, Rosen H, Boeve BF, Dacks P, Cosentino S, Abrahams S, Shneider N, Lingor P, Shefner J, Andersen PM, Al-Chalabi A, Turner MR; Attendees of the Second International Pre-Symptomatic ALS Workshop. Benatar M, et al. Nat Rev Neurol. 2024 May 20. doi: 10.1038/s41582-024-00961-z. Online ahead of print. Nat Rev Neurol. 2024. PMID: 38769202 Review.
Plasma metabolomic biomarker panel to distinguish patients with amyotrophic lateral sclerosis from disease mimics.
Lawton KA, Brown MV, Alexander D, Li Z, Wulff JE, Lawson R, Jaffa M, Milburn MV, Ryals JA, Bowser R, Cudkowicz ME, Berry JD; Northeast ALS Consortium. Lawton KA, et al. Amyotroph Lateral Scler Frontotemporal Degener. 2014 Sep;15(5-6):362-70. doi: 10.3109/21678421.2014.908311. Epub 2014 Jul 1. Amyotroph Lateral Scler Frontotemporal Degener. 2014. PMID: 24984169
A novel, efficient, randomized selection trial comparing combinations of drug therapy for ALS.
Gordon PH, Cheung YK, Levin B, Andrews H, Doorish C, Macarthur RB, Montes J, Bednarz K, Florence J, Rowin J, Boylan K, Mozaffar T, Tandan R, Mitsumoto H, Kelvin EA, Chapin J, Bedlack R, Rivner M, McCluskey LF, Pestronk A, Graves M, Sorenson EJ, Barohn RJ, Belsh JM, Lou JS, Levine T, Saperstein D, Miller RG, Scelsa SN; Combination Drug Selection Trial Study Group. Gordon PH, et al. Among authors: mozaffar t. Amyotroph Lateral Scler. 2008 Aug;9(4):212-22. doi: 10.1080/17482960802195632. Amyotroph Lateral Scler. 2008. PMID: 18608093 Free PMC article. Clinical Trial.
CAR T-Cell Therapy in Autoimmune Disease.
Howard JF Jr, Vu T, Mozaffar T. Howard JF Jr, et al. Among authors: mozaffar t. N Engl J Med. 2024 May 2;390(17):1629-1631. doi: 10.1056/NEJMc2403705. N Engl J Med. 2024. PMID: 38692299 No abstract available.
Debamestrocel multimodal effects on biomarker pathways in amyotrophic lateral sclerosis are linked to clinical outcomes.
Lindborg SR, Goyal NA, Katz J, Burford M, Li J, Kaspi H, Abramov N, Boulanger B, Berry JD, Nicholson K, Mozaffar T, Miller R, Jenkins L, Baloh RH, Lewis R, Staff NP, Owegi MA, Dagher B, Blondheim-Shraga NR, Gothelf Y, Levy YS, Kern R, Aricha R, Windebank AJ, Bowser R, Brown RH Jr, Cudkowicz ME. Lindborg SR, et al. Among authors: mozaffar t. Muscle Nerve. 2024 Jun;69(6):719-729. doi: 10.1002/mus.28093. Epub 2024 Apr 9. Muscle Nerve. 2024. PMID: 38593477 Clinical Trial.
272nd ENMC international workshop: 10 Years of progress - revision of the ENMC 2013 diagnostic criteria for inclusion body myositis and clinical trial readiness. 16-18 June 2023, Hoofddorp, The Netherlands.
Lilleker JB, Naddaf E, Saris CGJ, Schmidt J, de Visser M, Weihl CC; 272nd ENMC workshop participants. Lilleker JB, et al. Neuromuscul Disord. 2024 Apr;37:36-51. doi: 10.1016/j.nmd.2024.03.001. Epub 2024 Mar 7. Neuromuscul Disord. 2024. PMID: 38522330
Defining clinical endpoints in limb girdle muscular dystrophy: a GRASP-LGMD study.
Doody A, Alfano L, Diaz-Manera J, Lowes L, Mozaffar T, Mathews KD, Weihl CC, Wicklund M, Hung M, Statland J, Johnson NE; GRASP-LGMD Consortium. Doody A, et al. Among authors: mozaffar t. BMC Neurol. 2024 Mar 15;24(1):96. doi: 10.1186/s12883-024-03588-1. BMC Neurol. 2024. PMID: 38491364 Free PMC article.
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: mozaffar t. 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.
211 results