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Standardized Interoperable Data Collection for Myositis Research: Developing Expert Consensus on Common Data Elements for Myositis Outcome Measures. Arthritis Care Res (Hoboken). 2026 Mar 05. Saygin D, Diller M, Surampudi V, Bodkin M, Noroozi Farhadi P, Mecoli CA, Kessel A, Aggarwal R, Alexanderson H, Amato A, Bartels CM, Benveniste O, Best M, Chinoy H, Groot I, Feldman B, Huber AM, Kim H, Kim S, Kobert L, Leclair V, Lubinus M, Machado PM, Mammen A, McCann LJ, Mozaffar T, Oddis C, Paik JJ, Ravelli A, Ruperto N, Schmidt J, Werner E, Werth V, Schiffenbauer A, Scheuermann RH, Rider LG, IMACS Myositis CDE Working Group. PMID: 41787699.
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Miglustat: a first-in-class enzyme stabilizer for cipaglucosidase alfa for the treatment of late-onset Pompe disease. Ther Adv Rare Dis. 2026 Jan-Dec; 7:26330040261425686. Hopkin RJ, Byrne BJ, Dimachkie MM, Kishnani PS, Mozaffar T, Roberts M, Schoser B, van der Beek NAME, van der Ploeg AT, Wenninger S, Brudvig J, Fox B, Holdbrook F, Jain V, Johnson F, Zhang J, Parenti G. PMID: 41769220; PMCID: PMC12946408.
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Safety of home administration of cipaglucosidase alfa plus miglustat in late-onset Pompe disease: results from multiple clinical trials. Ther Adv Rare Dis. 2026 Jan-Dec; 7:26330040261416943. Andersen H, Díaz-Manera J, Goker-Alpan O, Mozaffar T, Sitaraman Das S, Fox B, Amon F, O'Brien-Prince K, Goldman M, Holdbrook F, Jain V, Byrne BJ. PMID: 41631150; PMCID: PMC12861377.
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BCMA-directed mRNA CAR T cell therapy for myasthenia gravis: a randomized, double-blind, placebo-controlled phase 2b trial. Nat Med. 2026 Jan 09. Vu T, Durmus H, Rivner M, Shroff S, Ragole T, Myers B, Pasnoor M, Small G, Karam C, Vullaganti M, Peltier A, Sahagian G, Feinberg MH, Slanksy A, Barnett-Tapia C, Siddiqi Z, Gwathmey K, Badruddoja MA, Kamboh H, Ruggerie RN, Fedak RR, Stewart CA, Kurtoglu M, Kalayoglu M, Singer M, Jewell CM, Miljkovic MD, Dimachkie M, Mozaffar T, Howard JF, MG-001 Study Team. PMID: 41514038.
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BCMA-directed mRNA CAR-T cell therapy for myasthenia gravis: exploratory biomarker analysis of a placebo-controlled phase 2b trial. Nat Med. 2026 Jan 09. Fedak RR, Ruggerie RN, Shan Y, Curvino EJ, de Sousa JF, Daniel S, Ngo-Casi M, Kamboh H, Vu T, Durmus H, Mozaffar T, Howard JF, English EP, Benson A, Duvernay MT, Singer MS, Kalayoglu MV, Brunn C, Bodansky A, Anderson MS, DeRisi JL, Garcia ST, Yu DJL, Zorn KC, Kurtoglu M, Miljkovic MD, Stewart CA, Jewell CM, MG-001 Study Team. PMID: 41514039.
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The Mythology of Polymyositis. Rheum Dis Clin North Am. 2025 11; 51(4):777-788. Biliciler S, Mozaffar T. PMID: 41161912.
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Myositis in 2025: Defining Subtypes, Closing Gaps, and Shaping the Future. Rheum Dis Clin North Am. 2025 Nov; 51(4):xvii-xviii. Paik JJ, Mozaffar T. PMID: 41161914.
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Consensus nomenclature and abbreviation for anti-synthetase syndrome: an IMACS project. Rheumatology (Oxford). 2025 Sep 01; 64(9):4930-4936. Aggarwal A, Chandra T, Keret S, Pauling JD, Shamim EA, Bonella F, Miller FW, Mammen A, Sambataro G, Liewluck T, Fernandez AP, Bartoloni E, Shinjo SK, Castañeda S, Werth V, Dimachkie MM, Katsumata Y, Campanilho-Marques R, Fujimoto M, Lilleker JB, Selva-O'Callaghan A, Mozaffar T, Gunawardena H, Mann H, Rojas Serrano J, Cavagna L, Moghadam-Kia S, Oddis CV, Ladha P, Mittal S, Haldule S, Edpuganti N, Sridhar S, Savaliya R, Batra V, Aggarwal R. PMID: 40286313; PMCID: PMC12407231.
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Durability of Response to B-Cell Maturation Antigen-Directed mRNA Cell Therapy in Myasthenia Gravis. Ann Clin Transl Neurol. 2025 Nov; 12(11):2358-2366. Chahin N, Sahagian G, Feinberg MH, Stewart CA, Jewell CM, Kurtoglu M, Miljkovic MD, Vu T, Mozaffar T, Howard JF, MG-001 Study Group. PMID: 40856088; PMCID: PMC12623823.
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Efficacy and safety of avalglucosidase alfa in patients with late-onset Pompe disease after 145 weeks of treatment during the COMET trial. J Neurol. 2025 Aug 16; 272(9):581. Kishnani PS, Díaz-Manera J, Illarioshkin S, van der Ploeg AT, Clemens PR, Day JW, Toscano A, Kushlaf H, Ladha S, Attarian S, Carvalho G, Kostera-Pruszczyk A, Erdem-Özdamar S, Goker-Alpan O, Mozaffar T, Straub V, Roberts M, Haack KA, Huynh-Ba O, Tammireddy S, Periquet M, Thibault N, Zhou T, Dimachkie MM, Schoser B, COMET Investigator Group. PMID: 40817977; PMCID: PMC12357813.
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Aging Accelerates Degradation of Human Neuromuscular Junction Following Peripheral Nerve Injury. Muscle Nerve. 2025 Nov; 72(5):1161-1167. Gonzales LP, Chen VY, Tedesco A, Andalib S, Lee C, Wright DJ, Johnston TR, Mozaffar T, Steward O, Gupta R. PMID: 41097942.
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Switching Enzyme Replacement Therapy for Late-Onset Pompe Disease From Alglucosidase Alfa to Cipaglucosidase Alfa Plus Miglustat: Post Hoc Effect Size Analysis of PROPEL. Muscle Nerve. 2025 Aug; 72(2):230-239. Kushlaf H, Díaz-Manera J, Bratkovic D, Byrne BJ, Claeys KG, Clemens PR, Dimachkie MM, Kishnani PS, Laforêt P, Roberts M, Schoser B, Toscano A, Castelli J, Holdbrook F, Sitaraman Das S, Goldman M, Mozaffar T, PROPEL Study Group. PMID: 40342075.
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Clinical Trial Readiness in Limb Girdle Muscular Dystrophy R1 (LGMDR1): A GRASP Consortium Study. Ann Clin Transl Neurol. 2025 Jun; 12(6):1179-1186. Hunn SM, Alfano LN, Jones A, Butler A, Lowes LP, Iammarino MA, Reash NF, Pietruszewski L, Sasidharan S, Currence M, Statland JM, Strahler T, Will R, Wicklund M, Dixon S, Augsburger R, Mozaffar T, Laubscher KM, Mockler SRH, Mathews KD, Stinson N, Leung DG, Stark MM, Horton RA, Kang PB, James MK, Clause A, Weihl CC, Johnson NE, GRASP-LGMD Consortium. PMID: 40237364; PMCID: PMC12172116.
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2024 VCP International Conference: Exploring multi-disciplinary approaches from basic science of valosin containing protein, an AAA+ ATPase protein, to the therapeutic advancement for VCP-associated multisystem proteinopathy. Neurobiol Dis. 2025 04; 207:106861. Peck A, Dadi A, Yavarow Z, Alfano LN, Anderson D, Arkin MR, Chou TF, D'Ambrosio ES, Diaz-Manera J, Dudley JP, Elder AG, Ghoshal N, Hart CE, Hart MM, Huryn DM, Johnson AE, Jones KB, Kimonis V, Kiskinis E, Lee EB, Lloyd TE, Mapstone M, Martin A, Meyer H, Mozaffar T, Onyike CU, Pfeffer G, Pindon A, Raman M, Richard I, Rubinsztein DC, Schiava M, Schütz AK, Shen PS, Southworth DR, Staffaroni AM, Taralio-Gravovac M, Weihl CC, Yao Q, Ye Y, Peck N. PMID: 40037468; PMCID: PMC11960434.
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Correction: 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). J Neurol. 2025 Feb 17; 272(3):213. 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. PMID: 39960520; PMCID: PMC11832724.
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Corrigendum: Cipaglucosidase alfa plus miglustat: linking mechanism of action to clinical outcomes in late-onset Pompe disease. Front Neurol. 2024; 15:1540452. 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. PMID: 39830206; PMCID: PMC11738947.
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Prospective observational study of FKRP-related limb-girdle muscular dystrophy R9: A GRASP consortium study. Ann Clin Transl Neurol. 2025 Feb; 12(2):332-344. Alfano LN, James MK, Grosfjeld Petersen K, Rudolf K, Vissing J, Augsburger R, Mozaffar T, Jones A, Butler A, Laubscher KM, Mockler SRH, Mathews KD, Iammarino MA, Reash NF, Pietruszewski L, Lowes LP, Strahler T, Wicklund M, Hunn S, Weihl CC, Sasidharan S, Currence M, Statland JM, Stinson N, Holzer M, Leung DG, Lott DJ, Kang PB, Holsten S, Desai U, Johnson NE, GRASP-LGMD Consortium. PMID: 39675022; PMCID: PMC11822816.
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The Dermatomyositis Disease Symptom Questionnaire (DM-DSQ): A Measure to Assess the Patient Experience of Dermatomyositis Symptoms. J Rheumatol. 2024 Dec 01; 51(12):1198-1207. Christopher-Stine L, Ciesluk A, Chinoy H, Goyal NA, Gunter K, Isenberg D, Kielhorn A, Lundberg IE, Mozaffar T, Rakhade S, Vandenberg G, Aggarwal R. PMID: 39089831.
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In response to: "Innovative cellular therapies for autoimmune diseases: expert-based position statement and clinical practice recommendations from the EBMT practice harmonization and guidelines committee" by Greco et al. EClinicalMedicine. 2024 Dec; 78:102918. Howard JF, Manzi SM, Miljkovic MD, Vu T, Mozaffar T. PMID: 39640933; PMCID: PMC11617738.
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Switching treatment to cipaglucosidase alfa plus miglustat positively affects patient-reported outcome measures in patients with late-onset Pompe disease. J Patient Rep Outcomes. 2024 Nov 13; 8(1):132. 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. PMID: 39535661; PMCID: PMC11561219.
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Efficacy and safety of maintenance intravenous immunoglobulin in generalized myasthenia gravis patients with acetylcholine receptor antibodies: A multicenter, double-blind, placebo-controlled trial. Muscle Nerve. 2025 Jan; 71(1):43-54. Bril V, Berkowicz T, Szczudlik A, Nicolle MW, Bednarik J, Hon P, Vaitkus A, Vu T, Rozsa C, Magnus T, Panczel G, Toomsoo T, Pasnoor M, Mozaffar T, Freimer M, Reuner U, Vécsei L, Souayah N, Levine T, Pascuzzi RM, Dalakas MC, Rivner M, Griffin R, Coll MQ, Mondou E. PMID: 39506903; PMCID: PMC11632570.
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Cipaglucosidase alfa plus miglustat: linking mechanism of action to clinical outcomes in late-onset Pompe disease. Front Neurol. 2024; 15:1451512. 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. PMID: 39494167; PMCID: PMC11527667.
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Comparing the efficacy of cipaglucosidase alfa plus miglustat with other enzyme replacement therapies for late-onset Pompe disease: a network meta-analysis utilizing patient-level and aggregate data. J Comp Eff Res. 2024 Oct; 13(10):e240045. Shohet S, Hummel N, Fu S, Keyzor I, MacCulloch A, Johnson N, Castelli J, Czarny-Ozga I, Mozaffar T, Thom H. PMID: 39287071; PMCID: PMC11426283.
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Case study of CD19 CAR T therapy in a subject with immune-mediate necrotizing myopathy treated in the RESET-Myositis phase I/II trial. Mol Ther. 2024 Nov 06; 32(11):3821-3828. Volkov J, Nunez D, Mozaffar T, Stadanlick J, Werner M, Vorndran Z, Ellis A, Williams J, Cicarelli J, Lam Q, Furmanak T, Schmitt C, Hadi-Nezhad F, Thompson D, Miller C, Little C, Chang D, Basu S. PMID: 39245937; PMCID: PMC11573600.
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Efficacy of avalglucosidase alfa on forced vital capacity percent predicted in treatment-naïve patients with late-onset Pompe disease: A pooled analysis of clinical trials. Mol Genet Metab Rep. 2024 Sep; 40:101109. Mozaffar T, Riou França L, Msihid J, Shukla P, Proskorovsky I, Zhou T, Periquet M, An Haack K, Pollissard L, Straub V. PMID: 39035044; PMCID: PMC11259910.
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CAR T-Cell Therapy in Autoimmune Disease. N Engl J Med. 2024 05 02; 390(17):1629-1631. Howard JF, Vu T, Mozaffar T. PMID: 38692299.
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Debamestrocel multimodal effects on biomarker pathways in amyotrophic lateral sclerosis are linked to clinical outcomes. Muscle Nerve. 2024 Jun; 69(6):719-729. 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, Cudkowicz ME. PMID: 38593477.
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Defining clinical endpoints in limb girdle muscular dystrophy: a GRASP-LGMD study. BMC Neurol. 2024 Mar 15; 24(1):96. 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. PMID: 38491364; PMCID: PMC10941356.
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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). J Neurol. 2024 May; 271(5):2810-2823. 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. PMID: 38418563; PMCID: PMC11055775.
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Recurring homozygous ACTN2 variant (p.Arg506Gly) causes a recessive myopathy. Ann Clin Transl Neurol. 2024 Mar; 11(3):629-640. Donkervoort S, Mohassel P, O'Leary M, Bonner DE, Hartley T, Acquaye N, Brull A, Mozaffar T, Saporta MA, Dyment DA, Sampson JB, Pajusalu S, Austin-Tse C, Hurth K, Cohen JS, McWalter K, Warman-Chardon J, Crunk A, Foley AR, Undiagnosed Diseases Network, Mammen AL, Wheeler MT, O'Donnell-Luria A, Bönnemann CG. PMID: 38311799; PMCID: PMC10963296.
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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). J Neurol. 2024 Apr; 271(4):1787-1801. 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. PMID: 38057636; PMCID: PMC10973052.
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Defining Clinical Endpoints in Limb Girdle Muscular Dystrophy: A GRASP-LGMD study. Res Sq. 2023 Oct 06. Doody A, Alfano L, Diaz-Manera J, Lowes L, Mozaffar T, Mathews K, Weihl CC, Wicklund M, Statland J, Johnson NE, GRASP-LGMD Consortium. PMID: 37886601; PMCID: PMC10602119.
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Safety and efficacy of arimoclomol for inclusion body myositis: a multicentre, randomised, double-blind, placebo-controlled trial. Lancet Neurol. 2023 10; 22(10):900-911. Machado PM, McDermott MP, Blaettler T, Sundgreen C, Amato AA, Ciafaloni E, Freimer M, Gibson SB, Jones SM, Levine TD, Lloyd TE, Mozaffar T, Shaibani AI, Wicklund M, Rosholm A, Carstensen TD, Bonefeld K, Jørgensen AN, Phonekeo K, Heim AJ, Herbelin L, Barohn RJ, Hanna MG, Dimachkie MM, Arimoclomol in IBM Investigator Team of the Neuromuscular Study Group. PMID: 37739573.
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Safety and outcomes with efgartigimod use for acetylcholine receptor-positive generalized myasthenia gravis in clinical practice. Muscle Nerve. 2023 Nov; 68(5):762-766. Katyal N, Halldorsdottir K, Govindarajan R, Shieh P, Muley S, Reyes P, Leung KK, Mullen J, Milani-Nejad S, Korb M, Goyal NA, Mozaffar T, Goyal N, Habib AA, Muppidi S. PMID: 37695277.
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Editorial: Inflammatory muscle diseases: an update. Front Neurol. 2023; 14:1259275. Tanboon J, Needham M, Mozaffar T, Stenzel W, Nishino I. PMID: 37614973; PMCID: PMC10442951.
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Single-cell and spatial transcriptomics identify a macrophage population associated with skeletal muscle fibrosis. Sci Adv. 2023 07 07; 9(27):eadd9984. Coulis G, Jaime D, Guerrero-Juarez C, Kastenschmidt JM, Farahat PK, Nguyen Q, Pervolarakis N, McLinden K, Thurlow L, Movahedi S, Hughes BS, Duarte J, Sorn A, Montoya E, Mozaffar I, Dragan M, Othy S, Joshi T, Hans CP, Kimonis V, MacLean AL, Nie Q, Wallace LM, Harper SQ, Mozaffar T, Hogarth MW, Bhattacharya S, Jaiswal JK, Golann DR, Su Q, Kessenbrock K, Stec M, Spencer MJ, Zamudio JR, Villalta SA. PMID: 37418531; PMCID: PMC10328414.
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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. Lancet Neurol. 2023 07; 22(7):578-590. Granit V, Benatar M, Kurtoglu M, Miljkovic MD, Chahin N, Sahagian G, Feinberg MH, Slansky A, Vu T, Jewell CM, Singer MS, Kalayoglu MV, Howard JF, Mozaffar T, MG-001 Study Team. PMID: 37353278; PMCID: PMC10416207.
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Effects of enzyme replacement therapy on bone density in late onset Pompe disease. Mol Genet Metab. 2023 11; 140(3):107644. Avanti M, Martin A, Columbres RC, Mozaffar T, Kimonis V. PMID: 37515933.
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Efficacy and Safety of Avalglucosidase Alfa in Patients With Late-Onset Pompe Disease After 97 Weeks: A Phase 3 Randomized Clinical Trial. JAMA Neurol. 2023 06 01; 80(6):558-567. 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. PMID: 37036722; PMCID: PMC10087094.
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Single-cell and spatial transcriptomics identify a macrophage population associated with skeletal muscle fibrosis. bioRxiv. 2023 Apr 18. Coulis G, Jaime D, Guerrero-Juarez C, Kastenschmidt JM, Farahat PK, Nguyen Q, Pervolarakis N, McLinden K, Thurlow L, Movahedi S, Duarte J, Sorn A, Montoya E, Mozaffar I, Dragan M, Othy S, Joshi T, Hans CP, Kimonis V, MacLean AL, Nie Q, Wallace LM, Harper SQ, Mozaffar T, Hogarth MW, Bhattacharya S, Jaiswal JK, Golann DR, Su Q, Kessenbrock K, Stec M, Spencer MJ, Zamudio JR, Villalta SA. PMID: 37131694; PMCID: PMC10153153.
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Provisional practice recommendation for the management of myopathy in VCP-associated multisystem proteinopathy. Ann Clin Transl Neurol. 2023 05; 10(5):686-695. 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. PMID: 37026610; PMCID: PMC10187720.
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Imaging beyond muscle magnetic resonance imaging in inclusion body myositis. Clin Exp Rheumatol. 2023 Mar; 41(2):386-392. Goyal NA, Mozaffar T, Dimachkie MM. PMID: 36700664.
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Randomized Double-Blind Placebo-Controlled Trial of the Corticosteroid-Sparing Effects of Immunoglobulin in Myasthenia Gravis. Neurology. 2023 02 14; 100(7):e671-e682. Bril V, Szczudlik A, Vaitkus A, Rozsa C, Kostera-Pruszczyk A, Hon P, Bednarik J, Tyblova M, Köhler W, Toomsoo T, Nowak RJ, Mozaffar T, Freimer ML, Nicolle MW, Magnus T, Pulley MT, Rivner M, Dimachkie MM, Distad BJ, Pascuzzi RM, Babiar D, Lin J, Querolt Coll M, Griffin R, Mondou E. PMID: 36270895; PMCID: PMC9969924.
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GNE myopathy: History, etiology, and treatment trials. Front Neurol. 2022; 13:1002310. Mullen J, Alrasheed K, Mozaffar T. PMID: 36330422; PMCID: PMC9623016.
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Inclusion body myositis: evolving concepts. Curr Opin Neurol. 2022 10 01; 35(5):604-610. Perez-Rosendahl M, Mozaffar T. PMID: 36069417; PMCID: PMC9472875.
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Facial Onset Sensory and Motor Neuronopathy-Like Syndrome: A Case Report. J Clin Neuromuscul Dis. 2022 Sep 01; 24(1):55-58. Bruss DM, Venkataraman P, Mozaffar T. PMID: 36005474.
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Correlations of disease severity outcome measures in inclusion body myositis. Neuromuscul Disord. 2022 10; 32(10):800-805. Goyal NA, Greenberg SA, Cauchi J, Araujo N, Li V, Wencel M, Irani T, Wang LH, Palma AM, Villalta SA, Mozaffar T. PMID: 36050251; PMCID: PMC10069380.
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Pompe Disease: a Clinical, Diagnostic, and Therapeutic Overview. Curr Treat Options Neurol. 2022 Nov; 24(11):573-588. Stevens D, Milani-Nejad S, Mozaffar T. PMID: 36969713; PMCID: PMC10035871.
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Utility of video-fundoscopy and prospects of portable stereo-photography of the ocular fundus in neurological patients. BMC Neurol. 2022 Feb 19; 22(1):61. Khachatryan T, Mozaffar T, Mnatsakanyan L. PMID: 35183131; PMCID: PMC8857737.
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Immunophenotyping of Inclusion Body Myositis Blood T and NK Cells. Neurology. 2022 03 29; 98(13):e1374-e1383. 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. PMID: 35131904; PMCID: PMC8967422.
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Development of a standard of care for patients with valosin-containing protein associated multisystem proteinopathy. Orphanet J Rare Dis. 2022 01 29; 17(1):23. Korb M, Peck A, Alfano LN, Berger KI, James MK, Ghoshal N, Healzer E, Henchcliffe C, Khan S, Mammen PPA, Patel S, Pfeffer G, Ralston SH, Roy B, Seeley WW, Swenson A, Mozaffar T, Weihl C, Kimonis V, VCP Standards of Care Working Group. PMID: 35093159; PMCID: PMC8800193.
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Loss of TDP-43 function and rimmed vacuoles persist after T cell depletion in a xenograft model of sporadic inclusion body myositis. Sci Transl Med. 2022 01 19; 14(628):eabi9196. Britson KA, Ling JP, Braunstein KE, Montagne JM, Kastenschmidt JM, Wilson A, Ikenaga C, Tsao W, Pinal-Fernandez I, Russell KA, Reed N, Mozaffar T, Wagner KR, Ostrow LW, Corse AM, Mammen AL, Villalta SA, Larman HB, Wong PC, Lloyd TE. PMID: 35044790; PMCID: PMC9118725.
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A randomized placebo-controlled phase 3 study of mesenchymal stem cells induced to secrete high levels of neurotrophic factors in amyotrophic lateral sclerosis. Muscle Nerve. 2022 03; 65(3):291-302. Cudkowicz ME, Lindborg SR, Goyal NA, Miller RG, Burford MJ, Berry JD, Nicholson KA, Mozaffar T, Katz JS, Jenkins LJ, Baloh RH, Lewis RA, Staff NP, Owegi MA, Berry DA, Gothelf Y, Levy YS, Aricha R, Kern RZ, Windebank AJ, Brown RH. PMID: 34890069; PMCID: PMC9305113.
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COVID-19 infection in patients with late-onset Pompe disease. Muscle Nerve. 2022 03; 65(3):334-336. Avelar J, Wencel M, Chumakova A, Mozaffar T. PMID: 34952985; PMCID: PMC8837697.
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Random forest: random results or meaningful insights for patients with facioscapulohumeral muscular dystrophy? Brain. 2021 12 16; 144(11):3288-3290. Alfano LN, Mozaffar T. PMID: 34636841; PMCID: PMC8677539.
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Safety and efficacy of avalglucosidase alfa versus alglucosidase alfa in patients with late-onset Pompe disease (COMET): a phase 3, randomised, multicentre trial. Lancet Neurol. 2021 12; 20(12):1012-1026. Diaz-Manera J, Kishnani PS, Kushlaf H, Ladha S, Mozaffar T, Straub V, Toscano A, van der Ploeg AT, Berger KI, Clemens PR, Chien YH, Day JW, Illarioshkin S, Roberts M, Attarian S, Borges JL, Bouhour F, Choi YC, Erdem-Ozdamar S, Goker-Alpan O, Kostera-Pruszczyk A, Haack KA, Hug C, Huynh-Ba O, Johnson J, Thibault N, Zhou T, Dimachkie MM, Schoser B, COMET Investigator Group. PMID: 34800399.
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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. Lancet Neurol. 2021 12; 20(12):1027-1037. 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. PMID: 34800400.
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Home-based gait analysis as an exploratory endpoint during a multicenter phase 1 trial in limb girdle muscular dystrophy type R2 and facioscapulohumeral muscular dystrophy. Muscle Nerve. 2022 02; 65(2):237-242. Gidaro T, Gasnier E, Annoussamy M, Vissing J, Attarian S, Mozaffar T, Iyadurai S, Wagner KR, Vissière D, Walker G, Shukla SS, Servais L. PMID: 34687225.
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Patient reported quality of life in limb girdle muscular dystrophy. Neuromuscul Disord. 2022 01; 32(1):57-64. Kovalchick LV, Bates K, Statland J, Weihl C, Kang PB, Lowes LP, Mozaffar T, Straub V, Wicklund M, Heatwole C, Johnson NE. PMID: 34961728; PMCID: PMC10311477.
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Investigating Late-Onset Pompe Prevalence in Neuromuscular Medicine Academic Practices: The IPaNeMA Study. Neurol Genet. 2021 Dec; 7(6):e623. 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. PMID: 36299500; PMCID: PMC9595038.
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Eculizumab in refractory generalized myasthenia gravis previously treated with rituximab: subgroup analysis of REGAIN and its extension study. Muscle Nerve. 2021 12; 64(6):662-669. Siddiqi ZA, Nowak RJ, Mozaffar T, O'Brien F, Yountz M, Patti F, REGAIN Study Group. PMID: 34590717.
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IgG regulation through FcRn blocking: A novel mechanism for the treatment of myasthenia gravis. J Neurol Sci. 2021 Nov 15; 430:118074. Wolfe GI, Ward ES, de Haard H, Ulrichts P, Mozaffar T, Pasnoor M, Vidarsson G. PMID: 34563918.
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Long-term efficacy of eculizumab in refractory generalized myasthenia gravis: responder analyses. Ann Clin Transl Neurol. 2021 07; 8(7):1398-1407. Howard JF, Karam C, Yountz M, O'Brien FL, Mozaffar T, REGAIN Study Group. PMID: 34043280; PMCID: PMC8283175.
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A stromal progenitor and ILC2 niche promotes muscle eosinophilia and fibrosis-associated gene expression. Cell Rep. 2021 04 13; 35(2):108997. Kastenschmidt JM, Coulis G, Farahat PK, Pham P, Rios R, Cristal TT, Mannaa AH, Ayer RE, Yahia R, Deshpande AA, Hughes BS, Savage AK, Giesige CR, Harper SQ, Locksley RM, Mozaffar T, Villalta SA. PMID: 33852849; PMCID: PMC8127948.
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Clinical utility of anti-cytosolic 5'-nucleotidase 1A antibody in idiopathic inflammatory myopathies. Ann Clin Transl Neurol. 2021 03; 8(3):571-578. Ikenaga C, Findlay AR, Goyal NA, Robinson S, Cauchi J, Hussain Y, Wang LH, Kershen JC, Beson BA, Wallendorf M, Bucelli RC, Mozaffar T, Pestronk A, Weihl CC. PMID: 33556224; PMCID: PMC7951108.
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Results from a 3-year Non-interventional, Observational Disease Monitoring Program in Adults with GNE Myopathy. J Neuromuscul Dis. 2021; 8(2):225-234. Lochmüller H, Behin A, Tournev I, Tarnopolsky M, Horváth R, Pogoryelova O, Shah J, Koutsoukos T, Skrinar A, Kakkis E, Bedrosian CL, Mozaffar T. PMID: 33459658; PMCID: PMC8075380.
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Multisystem proteinopathy: Where myopathy and motor neuron disease converge. Muscle Nerve. 2021 04; 63(4):442-454. Korb MK, Kimonis VE, Mozaffar T. PMID: 33145792.
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A phase 2, double-blinded, placebo-controlled trial of toll-like receptor 7/8/9 antagonist, IMO-8400, in dermatomyositis. J Am Acad Dermatol. 2021 Apr; 84(4):1160-1162. Kim YJ, Schiopu E, Dankó K, Mozaffar T, Chunduru S, Lees K, Goyal NA, Sarazin J, Fiorentino DF, Sarin KY. PMID: 32781178.
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Minimal manifestation status and prednisone withdrawal in the MGTX trial. Neurology. 2020 08 11; 95(6):e755-e766. Lee I, Kuo HC, Aban IB, Cutter GR, McPherson T, Kaminski HJ, Sussman J, Ströbel P, Oger J, Cea G, Heckmann JM, Evoli A, Nix W, Ciafaloni E, Antonini G, Witoonpanich R, King JO, Beydoun SR, Chalk CH, Barboi AC, Amato AA, Shaibani AI, Katirji B, Lecky BRF, Buckley C, Vincent A, Dias-Tosta E, Yoshikawa H, Waddington-Cruz M, Pulley MT, Rivner MH, Kostera-Pruszczyk A, Pascuzzi RM, Jackson CE, Verschuuren JJG, Massey JM, Kissel JT, Werneck LC, Benatar M, Barohn RJ, Tandan R, Mozaffar T, Conwit R, Minisman G, Sonett JR, Wolfe GI, MGTX study group. PMID: 32611638; PMCID: PMC7455358.
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Update on immune-mediated therapies for myasthenia gravis. Muscle Nerve. 2020 11; 62(5):579-592. Habib AA, Ahmadi Jazi G, Mozaffar T. PMID: 32462710.
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Clinical Effects of the Self-administered Subcutaneous Complement Inhibitor Zilucoplan in Patients With Moderate to Severe Generalized Myasthenia Gravis: Results of a Phase 2 Randomized, Double-Blind, Placebo-Controlled, Multicenter Clinical Trial. JAMA Neurol. 2020 05 01; 77(5):582-592. Howard JF, Nowak RJ, Wolfe GI, Freimer ML, Vu TH, Hinton JL, Benatar M, Duda PW, MacDougall JE, Farzaneh-Far R, Kaminski HJ, Zilucoplan MG Study Group, Barohn R, Dimachkie M, Pasnoor M, Farmakidis C, Liu T, Colgan S, Benatar MG, Bertorini T, Pillai R, Henegar R, Bromberg M, Gibson S, Janecki T, Freimer M, Elsheikh B, Matisak P, Genge A, Guidon A, David W, Habib AA, Mathew V, Mozaffar T, Hinton JL, Hewitt W, Barnett D, Sullivan P, Ho D, Howard JF, Traub RE, Chopra M, Kaminski HJ, Aly R, Bayat E, Abu-Rub M, Khan S, Lange D, Holzberg S, Khatri B, Lindman E, Olapo T, Sershon LM, Lisak RP, Bernitsas E, Jia K, Malik R, Lewis-Collins TD, Nicolle M, Nowak RJ, Sharma A, Roy B, Nye J, Pulley M, Berger A, Shabbir Y, Sachdev A, Patterson K, Siddiqi Z, Sivak M, Bratton J, Small G, Kohli A, Fetter M, Vu T, Lam L, Harvey B, Wolfe GI, Silvestri N, Patrick K, Zakalik K, Duda PW, MacDougall J, Farzaneh-Far R, Pontius A, Hoarty M. PMID: 32065623; PMCID: PMC7042797.
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Plasma creatinine and oxidative stress biomarkers in amyotrophic lateral sclerosis. Amyotroph Lateral Scler Frontotemporal Degener. 2020 05; 21(3-4):263-272. Mitsumoto H, Garofalo DC, Santella RM, Sorenson EJ, Oskarsson B, Fernandes JAM, Andrews H, Hupf J, Gilmore M, Heitzman D, Bedlack RS, Katz JS, Barohn RJ, Kasarskis EJ, Lomen-Hoerth C, Mozaffar T, Nations SP, Swenson AJ, Factor-Litvak P. PMID: 32276554; PMCID: PMC7373369.
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Corrigendum to "Longitudinal Screening Detects Cognitive Stability and Behavioral Deterioration in ALS Patients". Behav Neurol. 2019; 2019:6704740. Woolley S, Goetz R, Factor-Litvak P, Murphy J, Hupf J, Garofalo DC, Lomen-Hoerth C, Andrews H, Heitzman D, Bedlack R, Katz J, Barohn R, Sorenson E, Oskarsson B, Filho AF, Kasarskis E, Mozaffar T, Nations S, Swenson A, Koczon-Jaremko A, Christodoulou G, Mitsumoto H. PMID: 31885727; PMCID: PMC6914922.
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Oculopharyngeal Muscular Dystrophy, an Often Misdiagnosed Neuromuscular Disorder: A Southern California Experience. J Clin Neuromuscul Dis. 2019 Dec; 21(2):61-68. Goyal NA, Mozaffar T, Chui LA. PMID: 31743248.
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QuantiMus: A Machine Learning-Based Approach for High Precision Analysis of Skeletal Muscle Morphology. Front Physiol. 2019; 10:1416. Kastenschmidt JM, Ellefsen KL, Mannaa AH, Giebel JJ, Yahia R, Ayer RE, Pham P, Rios R, Vetrone SA, Mozaffar T, Villalta SA. PMID: 31849692; PMCID: PMC6895564.
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Adult MTM1-related myopathy carriers: Classification based on deep phenotyping. Neurology. 2019 10 15; 93(16):e1535-e1542. Cocanougher BT, Flynn L, Yun P, Jain M, Waite M, Vasavada R, Wittenbach JD, de Chastonay S, Chhibber S, Innes AM, MacLaren L, Mozaffar T, Arai AE, Donkervoort S, Bönnemann CG, Foley AR. PMID: 31541013; PMCID: PMC6815204.
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Limb-girdle muscular dystrophy: A perspective from adult patients on what matters most. Muscle Nerve. 2019 10; 60(4):419-424. Hunter M, Heatwole C, Wicklund M, Weihl CC, Mozaffar T, Statland JM, Johnson NE. PMID: 31298728.
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Reader response: Pearls & Oy-sters: Pembrolizumab-induced myasthenia gravis. Neurology. 2019 07 23; 93(4):183-184. Robbins NM, Mozaffar T, Mammen AL, Liewluck T, Guidon A, Lawson VH. PMID: 31332091.
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New family with HSPB8-associated autosomal dominant rimmed vacuolar myopathy. Neurol Genet. 2019 Aug; 5(4):e349. Al-Tahan S, Weiss L, Yu H, Tang S, Saporta M, Vihola A, Mozaffar T, Udd B, Kimonis V. PMID: 31403083; PMCID: PMC6659134.
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Variable clinical features and genotype-phenotype correlations in 18 patients with late-onset Pompe disease. Ann Transl Med. 2019 Jul; 7(13):276. Alandy-Dy J, Wencel M, Hall K, Simon J, Chen Y, Valenti E, Yang J, Bali D, Lakatos A, Goyal N, Mozaffar T, Kimonis V. PMID: 31392188; PMCID: PMC6642945.
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SMCHD1 mutation spectrum for facioscapulohumeral muscular dystrophy type 2 (FSHD2) and Bosma arhinia microphthalmia syndrome (BAMS) reveals disease-specific localisation of variants in the ATPase domain. J Med Genet. 2019 10; 56(10):693-700. Lemmers RJLF, van der Stoep N, Vliet PJV, Moore SA, San Leon Granado D, Johnson K, Topf A, Straub V, Evangelista T, Mozaffar T, Kimonis V, Shaw ND, Selvatici R, Ferlini A, Voermans N, van Engelen B, Sacconi S, Tawil R, Lamers M, van der Maarel SM. PMID: 31243061; PMCID: PMC6800092.
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Randomized phase 2 study of FcRn antagonist efgartigimod in generalized myasthenia gravis. Neurology. 2019 06 04; 92(23):e2661-e2673. Howard JF, Bril V, Burns TM, Mantegazza R, Bilinska M, Szczudlik A, Beydoun S, Garrido FJRR, Piehl F, Rottoli M, Van Damme P, Vu T, Evoli A, Freimer M, Mozaffar T, Ward ES, Dreier T, Ulrichts P, Verschueren K, Guglietta A, de Haard H, Leupin N, Verschuuren JJGM, Efgartigimod MG Study Group. PMID: 31118245; PMCID: PMC6556100.
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Homozygosity for the A431E mutation in PSEN1 presenting with a relatively aggressive phenotype. Neurosci Lett. 2019 04 23; 699:195-198. Parker J, Mozaffar T, Messmore A, Deignan JL, Kimonis VE, Ringman JM. PMID: 30716424; PMCID: PMC7759143.
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A phase 3 randomized study evaluating sialic acid extended-release for GNE myopathy. Neurology. 2019 04 30; 92(18):e2109-e2117. Lochmüller H, Behin A, Caraco Y, Lau H, Mirabella M, Tournev I, Tarnopolsky M, Pogoryelova O, Woods C, Lai A, Shah J, Koutsoukos T, Skrinar A, Mansbach H, Kakkis E, Mozaffar T. PMID: 31036580; PMCID: PMC6512882.
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Long-term effect of thymectomy plus prednisone versus prednisone alone in patients with non-thymomatous myasthenia gravis: 2-year extension of the MGTX randomised trial. Lancet Neurol. 2019 03; 18(3):259-268. Wolfe GI, Kaminski HJ, Aban IB, Minisman G, Kuo HC, Marx A, Ströbel P, Mazia C, Oger J, Cea JG, Heckmann JM, Evoli A, Nix W, Ciafaloni E, Antonini G, Witoonpanich R, King JO, Beydoun SR, Chalk CH, Barboi AC, Amato AA, Shaibani AI, Katirji B, Lecky BRF, Buckley C, Vincent A, Dias-Tosta E, Yoshikawa H, Waddington-Cruz M, Pulley MT, Rivner MH, Kostera-Pruszczyk A, Pascuzzi RM, Jackson CE, Verschuuren JJGM, Massey JM, Kissel JT, Werneck LC, Benatar M, Barohn RJ, Tandan R, Mozaffar T, Silvestri NJ, Conwit R, Sonett JR, Jaretzki A, Newsom-Davis J, Cutter GR, MGTX Study Group. PMID: 30692052; PMCID: PMC6774753.
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Expanding the importance of HMERF titinopathy: new mutations and clinical aspects. J Neurol. 2019 Mar; 266(3):680-690. Palmio J, Leonard-Louis S, Sacconi S, Savarese M, Penttilä S, Semmler AL, Kress W, Mozaffar T, Lai T, Stojkovic T, Berardo A, Reisin R, Attarian S, Urtizberea A, Cobo AM, Maggi L, Kurbatov S, Nikitin S, Milisenda JC, Fatehi F, Raimondi M, Silveira F, Hackman P, Claeys KG, Udd B. PMID: 30666435; PMCID: PMC6394805.
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Genetic landscape and novel disease mechanisms from a large LGMD cohort of 4656 patients. Ann Clin Transl Neurol. 2018 Dec; 5(12):1574-1587. Nallamilli BRR, Chakravorty S, Kesari A, Tanner A, Ankala A, Schneider T, da Silva C, Beadling R, Alexander JJ, Askree SH, Whitt Z, Bean L, Collins C, Khadilkar S, Gaitonde P, Dastur R, Wicklund M, Mozaffar T, Harms M, Rufibach L, Mittal P, Hegde M. PMID: 30564623; PMCID: PMC6292381.
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Rasagiline for amyotrophic lateral sclerosis: A randomized, controlled trial. Muscle Nerve. 2019 02; 59(2):201-207. Statland JM, Moore D, Wang Y, Walsh M, Mozaffar T, Elman L, Nations SP, Mitsumoto H, Fernandes JA, Saperstein D, Hayat G, Herbelin L, Karam C, Katz J, Wilkins HM, Agbas A, Swerdlow RH, Santella RM, Dimachkie MM, Barohn RJ, Rasagiline Investigators of the Muscle Study Group and Western ALS Consortium. PMID: 30192007; PMCID: PMC6545236.
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Longitudinal Screening Detects Cognitive Stability and Behavioral Deterioration in ALS Patients. Behav Neurol. 2018; 2018:5969137. Woolley S, Goetz R, Factor-Litvak P, Murphy J, Hupf J, Garofalo DC, Lomen-Hoerth C, Andrews H, Heitzman D, Bedlack R, Katz J, Barohn R, Sorenson E, Oskarsson B, Filho AF, Kasarskis E, Mozaffar T, Nations S, Swenson A, Koczon-Jaremko A, Christodoulou G, Mitsumoto H. PMID: 30515252; PMCID: PMC6234441.
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Safety, tolerability, and preliminary efficacy of an IGF-1 mimetic in patients with spinal and bulbar muscular atrophy: a randomised, placebo-controlled trial. Lancet Neurol. 2018 12; 17(12):1043-1052. Grunseich C, Miller R, Swan T, Glass DJ, El Mouelhi M, Fornaro M, Petricoul O, Vostiar I, Roubenoff R, Meriggioli MN, Kokkinis A, Guber RD, Budron MS, Vissing J, Soraru G, Mozaffar T, Ludolph A, Kissel JT, Fischbeck KH, BVS857 study group. PMID: 30337273; PMCID: PMC6415539.
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Review process for IVIg treatment: Lessons learned from INSIGHTS neuropathy study. Neurol Clin Pract. 2018 Oct; 8(5):429-436. Levine TD, Katz JS, Barohn R, Vaughan LJ, Dimachkie MM, Saperstein DS, Mozaffar T, Wolfe GI, Mayo MS, Badger GJ, Katzin L, Ritt E, Greer M, DiStefano J, Schmidt PM. PMID: 30564497; PMCID: PMC6276327.
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Novel Therapeutic Options in Treatment of Idiopathic Inflammatory Myopathies. Curr Treat Options Neurol. 2018 Jul 23; 20(9):37. Goyal NA, Mozaffar T. PMID: 30033499.
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A cross-sectional analysis of clinical evaluation in 35 individuals with mutations of the valosin-containing protein gene. Neuromuscul Disord. 2018 09; 28(9):778-786. Plewa J, Surampalli A, Wencel M, Milad M, Donkervoort S, Caiozzo VJ, Goyal N, Mozaffar T, Kimonis V. PMID: 30097247; PMCID: PMC6490182.
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Mexiletine for muscle cramps in amyotrophic lateral sclerosis: A randomized, double-blind crossover trial. Muscle Nerve. 2018 Mar 06. Oskarsson B, Moore D, Mozaffar T, Ravits J, Wiedau-Pazos M, Parziale N, Joyce NC, Mandeville R, Goyal N, Cudkowicz ME, Weiss M, Miller RG, McDonald CM. PMID: 29510461; PMCID: PMC6126993.
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Genotype-phenotype study in patients with valosin-containing protein mutations associated with multisystem proteinopathy. Clin Genet. 2018 Jan; 93(1):119-125. Al-Obeidi E, Al-Tahan S, Surampalli A, Goyal N, Wang AK, Hermann A, Omizo M, Smith C, Mozaffar T, Kimonis V. PMID: 28692196; PMCID: PMC5739971.
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Duvoglustat HCl Increases Systemic and Tissue Exposure of Active Acid α-Glucosidase in Pompe Patients Co-administered with Alglucosidase α. Mol Ther. 2017 05 03; 25(5):1199-1208. Kishnani P, Tarnopolsky M, Roberts M, Sivakumar K, Dasouki M, Dimachkie MM, Finanger E, Goker-Alpan O, Guter KA, Mozaffar T, Pervaiz MA, Laforet P, Levine T, Adera M, Lazauskas R, Sitaraman S, Khanna R, Benjamin E, Feng J, Flanagan JJ, Barth J, Barlow C, Lockhart DJ, Valenzano KJ, Boudes P, Johnson FK, Byrne B. PMID: 28341561; PMCID: PMC5417791.
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Sporadic inclusion body myositis misdiagnosed as idiopathic granulomatous myositis. Neuromuscul Disord. 2016 11; 26(11):741-743. Lavian M, Goyal N, Mozaffar T. PMID: 27692540.
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Randomized Trial of Thymectomy in Myasthenia Gravis. N Engl J Med. 2016 08 11; 375(6):511-22. Wolfe GI, Kaminski HJ, Aban IB, Minisman G, Kuo HC, Marx A, Ströbel P, Mazia C, Oger J, Cea JG, Heckmann JM, Evoli A, Nix W, Ciafaloni E, Antonini G, Witoonpanich R, King JO, Beydoun SR, Chalk CH, Barboi AC, Amato AA, Shaibani AI, Katirji B, Lecky BR, Buckley C, Vincent A, Dias-Tosta E, Yoshikawa H, Waddington-Cruz M, Pulley MT, Rivner MH, Kostera-Pruszczyk A, Pascuzzi RM, Jackson CE, Garcia Ramos GS, Verschuuren JJ, Massey JM, Kissel JT, Werneck LC, Benatar M, Barohn RJ, Tandan R, Mozaffar T, Conwit R, Odenkirchen J, Sonett JR, Jaretzki A, Newsom-Davis J, Cutter GR, MGTX Study Group. PMID: 27509100; PMCID: PMC5189669.
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Critically re-evaluating a common technique: Accuracy, reliability, and confirmation bias of EMG. Neurology. 2016 08 09; 87(6):645. Charles JA, Souayah N, Narayanaswami P, Jones L, Weiss M, Mozaffar T, Rutkove SB. PMID: 27502964.
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A randomized controlled trial of methotrexate for patients with generalized myasthenia gravis. Neurology. 2016 Jul 05; 87(1):57-64. Pasnoor M, He J, Herbelin L, Burns TM, Nations S, Bril V, Wang AK, Elsheikh BH, Kissel JT, Saperstein D, Shaibani JA, Jackson C, Swenson A, Howard JF, Goyal N, David W, Wicklund M, Pulley M, Becker M, Mozaffar T, Benatar M, Pazcuzzi R, Simpson E, Rosenfeld J, Dimachkie MM, Statland JM, Barohn RJ, Methotrexate in MG Investigators of the Muscle Study Group. PMID: 27306628; PMCID: PMC4932232.
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Defining SOD1 ALS natural history to guide therapeutic clinical trial design. J Neurol Neurosurg Psychiatry. 2017 Feb; 88(2):99-105. Bali T, Self W, Liu J, Siddique T, Wang LH, Bird TD, Ratti E, Atassi N, Boylan KB, Glass JD, Maragakis NJ, Caress JB, McCluskey LF, Appel SH, Wymer JP, Gibson S, Zinman L, Mozaffar T, Callaghan B, McVey AL, Jockel-Balsarotti J, Allred P, Fisher ER, Lopate G, Pestronk A, Cudkowicz ME, Miller TM. PMID: 27261500; PMCID: PMC5136332.
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Prospective exploratory muscle biopsy, imaging, and functional assessment in patients with late-onset Pompe disease treated with alglucosidase alfa: The EMBASSY Study. Mol Genet Metab. 2016 09; 119(1-2):115-23. van der Ploeg A, Carlier PG, Carlier RY, Kissel JT, Schoser B, Wenninger S, Pestronk A, Barohn RJ, Dimachkie MM, Goker-Alpan O, Mozaffar T, Pena LD, Simmons Z, Straub V, Guglieri M, Young P, Boentert M, Baudin PY, Wens S, Shafi R, Bjartmar C, Thurberg BL. PMID: 27473031.
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Mortality and Causes of Death in Patients with Sporadic Inclusion Body Myositis: Survey Study Based on the Clinical Experience of Specialists in Australia, Europe and the USA. J Neuromuscul Dis. 2016 03 03; 3(1):67-75. Price MA, Barghout V, Benveniste O, Christopher-Stine L, Corbett A, de Visser M, Hilton-Jones D, Kissel JT, Lloyd TE, Lundberg IE, Mastaglia F, Mozaffar T, Needham M, Schmidt J, Sivakumar K, DeMuro C, Tseng BS. PMID: 27854208; PMCID: PMC5271419.
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Cognitive-behavioral screening reveals prevalent impairment in a large multicenter ALS cohort. Neurology. 2016 Mar 01; 86(9):813-20. Murphy J, Factor-Litvak P, Goetz R, Lomen-Hoerth C, Nagy PL, Hupf J, Singleton J, Woolley S, Andrews H, Heitzman D, Bedlack RS, Katz JS, Barohn RJ, Sorenson EJ, Oskarsson B, Fernandes Filho JA, Kasarskis EJ, Mozaffar T, Rollins YD, Nations SP, Swenson AJ, Koczon-Jaremko BA, Mitsumoto H, ALS COSMOS. PMID: 26802094; PMCID: PMC4793785.
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Critically re-evaluating a common technique: Accuracy, reliability, and confirmation bias of EMG. Neurology. 2016 Jan 19; 86(3):218-23. Narayanaswami P, Geisbush T, Jones L, Weiss M, Mozaffar T, Gronseth G, Rutkove SB. PMID: 26701380; PMCID: PMC4733154.
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Editorial by concerned physicians: Unintended effect of the orphan drug act on the potential cost of 3,4-diaminopyridine. Muscle Nerve. 2016 Feb; 53(2):165-8. Burns TM, Smith GA, Allen JA, Amato AA, Arnold WD, Barohn R, Benatar M, Bird SJ, Bromberg M, Chahin N, Ciafaloni E, Cohen JA, Corse A, Crum BA, David WS, Dimberg E, Sousa EA, Donofrio PD, Dyck PJ, Engel AG, Ensrud ER, Ferrante M, Freimer M, Gable KL, Gibson S, Gilchrist JM, Goldstein JM, Gooch CL, Goodman BP, Gorelov D, Gospe SM, Goyal NA, Guidon AC, Guptill JT, Gutmann L, Gutmann L, Gwathmey K, Harati Y, Harper CM, Hehir MK, Hobson-Webb LD, Howard JF, Jackson CE, Johnson N, Jones SM, Juel VC, Kaminski HJ, Karam C, Kennelly KD, Khella S, Khoury J, Kincaid JC, Kissel JT, Kolb N, Lacomis D, Ladha S, Larriviere D, Lewis RA, Li Y, Litchy WJ, Logigian E, Lou JS, MacGowen DJ, Maselli R, Massey JM, Mauermann ML, Mathews KD, Meriggioli MN, Miller RG, Moon JS, Mozaffar T, Nations SP, Nowak RJ, Ostrow LW, Pascuzzi RM, Peltier A, Ruzhansky K, Richman DP, Ross MA, Rubin DI, Russell JA, Sachs GM, Salajegheh MK, Saperstein DS, Scelsa S, Selcen D, Shaibani A, Shieh PB, Silvestri NJ, Singleton JR, Smith BE, So YT, Solorzano G, Sorenson EJ, Srinivasen J, Tavee J, Tawil R, Thaisetthawatkul P, Thornton C, Trivedi J, Vernino S, Wang AK, Webb TA, Weiss MD, Windebank AJ, Wolfe GI. PMID: 26662952.
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Pulmonary function tests (maximum inspiratory pressure, maximum expiratory pressure, vital capacity, forced vital capacity) predict ventilator use in late-onset Pompe disease. Neuromuscul Disord. 2016 Feb; 26(2):136-45. Johnson EM, Roberts M, Mozaffar T, Young P, Quartel A, Berger KI. PMID: 26794303.
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GMPPB-Associated Dystroglycanopathy: Emerging Common Variants with Phenotype Correlation. Hum Mutat. 2015 Dec; 36(12):1159-63. Jensen BS, Willer T, Saade DN, Cox MO, Mozaffar T, Scavina M, Stefans VA, Winder TL, Campbell KP, Moore SA, Mathews KD. PMID: 26310427; PMCID: PMC4843780.
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Tongue atrophy and fasciculations in transthyretin familial amyloid neuropathy: An ALS mimicker. Neurol Genet. 2015 Aug; 1(2):e18. Goyal NA, Mozaffar T. PMID: 27066555; PMCID: PMC4807904.
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Diagnostic value of MRI in inflammatory myositis. Neurol Neuroimmunol Neuroinflamm. 2015 Aug; 2(4):e128. Lai TT, Mozaffar T. PMID: 26185775; PMCID: PMC4503409.
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An overview of polymyositis and dermatomyositis. Muscle Nerve. 2015 May; 51(5):638-56. Findlay AR, Goyal NA, Mozaffar T. PMID: 25641317.
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Desert hedgehog is a mediator of demyelination in compression neuropathies. Exp Neurol. 2015 Sep; 271:84-94. Jung J, Frump D, Su J, Wang W, Mozaffar T, Gupta R. PMID: 25936873.
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Seropositivity for NT5c1A antibody in sporadic inclusion body myositis predicts more severe motor, bulbar and respiratory involvement. J Neurol Neurosurg Psychiatry. 2016 Apr; 87(4):373-8. Goyal NA, Cash TM, Alam U, Enam S, Tierney P, Araujo N, Mozaffar FH, Pestronk A, Mozaffar T. PMID: 25857661; PMCID: PMC6192256.
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Activation of the Wnt/β-catenin signaling cascade after traumatic nerve injury. Neuroscience. 2015 May 21; 294:101-8. Kurimoto S, Jung J, Tapadia M, Lengfeld J, Agalliu D, Waterman M, Mozaffar T, Gupta R. PMID: 25743255; PMCID: PMC5384639.
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Topical tranexamic Acid does not affect electrophysiologic or neurovascular sciatic nerve markers in an animal model. Clin Orthop Relat Res. 2015 Mar; 473(3):1074-82. Schwarzkopf R, Dang P, Luu M, Mozaffar T, Gupta R. PMID: 25560955; PMCID: PMC4317440.
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A Phase 4 Prospective Study in Patients with Adult Pompe Disease Treated with Alglucosidase Alfa. J Neuromuscul Dis. 2015; 2(s1):S72-S73. Thurberg BL, Carlier P, Kissel JT, Schoser B, Pestronk A, Barohn RJ, Goker-Alpan O, Mozaffar T, Pena LD, Simmons Z, Straub V, Young P, Shafi R, Bjartmar C, van der Ploeg A. PMID: 27858658.
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Experimental trials in amyotrophic lateral sclerosis: a review of recently completed, ongoing and planned trials using existing and novel drugs. Expert Opin Investig Drugs. 2014 Nov; 23(11):1541-51. Goyal NA, Mozaffar T. PMID: 24965719.
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Early Surgical Decompression Restores Neurovascular Blood Flow and Ischemic Parameters in an in Vivo Animal Model of Nerve Compression Injury. J Bone Joint Surg Am. 2014 Jun 04; 96(11):897-906. Jung J, Hahn P, Choi B, Mozaffar T, Gupta R. PMID: 24897737; PMCID: PMC4049242.
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Patient characteristics and comorbidities associated with cerebrovascular accident following acute myocardial infarction in the United States. Int J Cardiol. 2014 Aug 01; 175(2):323-7. Naderi N, Masoomi H, Mozaffar T, Malik S. PMID: 24874908; PMCID: PMC4121439.
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Hypercaloric enteral nutrition in patients with amyotrophic lateral sclerosis: a randomised, double-blind, placebo-controlled phase 2 trial. Lancet. 2014 Jun 14; 383(9934):2065-2072. Wills AM, Hubbard J, Macklin EA, Glass J, Tandan R, Simpson EP, Brooks B, Gelinas D, Mitsumoto H, Mozaffar T, Hanes GP, Ladha SS, Heiman-Patterson T, Katz J, Lou JS, Mahoney K, Grasso D, Lawson R, Yu H, Cudkowicz M, MDA Clinical Research Network. PMID: 24582471; PMCID: PMC4176708.
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ALS Multicenter Cohort Study of Oxidative Stress (ALS COSMOS): study methodology, recruitment, and baseline demographic and disease characteristics. Amyotroph Lateral Scler Frontotemporal Degener. 2014 Jun; 15(3-4):192-203. Mitsumoto H, Factor-Litvak P, Andrews H, Goetz RR, Andrews L, Rabkin JG, McElhiney M, Nieves J, Santella RM, Murphy J, Hupf J, Singleton J, Merle D, Kilty M, Heitzman D, Bedlack RS, Miller RG, Katz JS, Forshew D, Barohn RJ, Sorenson EJ, Oskarsson B, Fernandes Filho JA, Kasarskis EJ, Lomen-Hoerth C, Mozaffar T, Rollins YD, Nations SP, Swenson AJ, Shefner JM, Andrews JA, Koczon-Jaremko BA, ALS COSMOS Study Group. PMID: 24564738; PMCID: PMC4310702.
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Respiratory and nutritional support in amyotrophic lateral sclerosis. Curr Treat Options Neurol. 2014 Feb; 16(2):270. Goyal NA, Mozaffar T. PMID: 24390801.
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Lipid-enriched diet rescues lethality and slows down progression in a murine model of VCP-associated disease. Hum Mol Genet. 2014 Mar 01; 23(5):1333-44. Llewellyn KJ, Nalbandian A, Jung KM, Nguyen C, Avanesian A, Mozaffar T, Piomelli D, Kimonis VE. PMID: 24158850; PMCID: PMC3919004.
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ALS Untangled No. 20: the Deanna protocol. Amyotroph Lateral Scler Frontotemporal Degener. 2013 May; 14(4):319-23. ALSUntangled Group, Fournier C, Bedlack B, Hardiman O, Heiman-Patterson T, Gutmann L, Bromberg M, Ostrow L, Carter G, Kabashi E, Bertorini T, Mozaffar T, Andersen P, Dietz J, Gamez J, Dimachkie M, Wang Y, Wicks P, Heywood J, Novella S, Rowland LP, Pioro E, Kinsley L, Mitchell K, Glass J, Sathornsumetee S, Kwiecinski H, Baker J, Atassi N, Forshew D, Ravits J, Conwit R, Jackson C, Sherman A, Dalton K, Tindall K, Gonzalez G, Robertson J, Phillips L, Benatar M, Sorenson E, Shoesmith C, Nash S, Maragakis N, Moore D, Caress J, Boylan K, Armon C, Grosso M, Gerecke B, Wymer J, Oskarsson B, Bowser R, Drory V, Shefner J, Lechtzin N, Leitner M, Miller R, Mitsumoto H, Levine T, Russell J, Sharma K, Saperstein D, McClusky L, MacGowan D, Licht J, Verma A, Strong M, Lomen-Hoerth C, Tandan R, Rivner M, Kolb S, Polak M, Rudnicki S, Kittrell P, Quereshi M, Sachs G, Pattee G, Weiss M, Kissel J, Goldstein J, Rothstein J, Pastula D, Gleb L, Ogino M, Rosenfeld J, Carmi E, Oster C, Barkhaus P, Valor E. PMID: 23638638.
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A randomized, double-blind, placebo-controlled phase II study of eculizumab in patients with refractory generalized myasthenia gravis. Muscle Nerve. 2013 Jul; 48(1):76-84. Howard JF, Barohn RJ, Cutter GR, Freimer M, Juel VC, Mozaffar T, Mellion ML, Benatar MG, Farrugia ME, Wang JJ, Malhotra SS, Kissel JT, MG Study Group. PMID: 23512355.
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Novel GNE mutations in autosomal recessive hereditary inclusion body myopathy patients. Genet Test Mol Biomarkers. 2013 May; 17(5):376-82. No D, Valles-Ayoub Y, Carbajo R, Khokher Z, Sandoval L, Stein B, Tarnopolsky MA, Mozaffar T, Darvish B, Pietruszka M, Darvish D. PMID: 23437777.
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A Review of Spasticity Treatments: Pharmacological and Interventional Approaches. Crit Rev Phys Rehabil Med. 2013; 25(1-2):11-22. Chang E, Ghosh N, Yanni D, Lee S, Alexandru D, Mozaffar T. PMID: 25750484; PMCID: PMC4349402.
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Matrix metalloproteinase 3 deletion preserves denervated motor endplates after traumatic nerve injury. Ann Neurol. 2013 Feb; 73(2):210-23. Chao T, Frump D, Lin M, Caiozzo VJ, Mozaffar T, Steward O, Gupta R. PMID: 23281061.
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A progressive translational mouse model of human valosin-containing protein disease: the VCP(R155H/+) mouse. Muscle Nerve. 2013 Feb; 47(2):260-70. Nalbandian A, Llewellyn KJ, Badadani M, Yin HZ, Nguyen C, Katheria V, Watts G, Mukherjee J, Vesa J, Caiozzo V, Mozaffar T, Weiss JH, Kimonis VE. PMID: 23169451; PMCID: PMC3556223.
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Genotype-phenotype studies of VCP-associated inclusion body myopathy with Paget disease of bone and/or frontotemporal dementia. Clin Genet. 2013 May; 83(5):422-31. Mehta SG, Khare M, Ramani R, Watts GD, Simon M, Osann KE, Donkervoort S, Dec E, Nalbandian A, Platt J, Pasquali M, Wang A, Mozaffar T, Smith CD, Kimonis VE. PMID: 22909335; PMCID: PMC3618576.
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The homozygote VCP(R¹⁵⁵H/R¹⁵⁵H) mouse model exhibits accelerated human VCP-associated disease pathology. PLoS One. 2012; 7(9):e46308. Nalbandian A, Llewellyn KJ, Kitazawa M, Yin HZ, Badadani M, Khanlou N, Edwards R, Nguyen C, Mukherjee J, Mozaffar T, Watts G, Weiss J, Kimonis VE. PMID: 23029473; PMCID: PMC3460820.
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Slow development of ALS-like spinal cord pathology in mutant valosin-containing protein gene knock-in mice. Cell Death Dis. 2012 Aug 16; 3:e374. Yin HZ, Nalbandian A, Hsu CI, Li S, Llewellyn KJ, Mozaffar T, Kimonis VE, Weiss JH. PMID: 22898872; PMCID: PMC3434652.
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Chronic nerve compression alters Schwann cell myelin architecture in a murine model. Muscle Nerve. 2012 Feb; 45(2):231-41. Gupta R, Nassiri N, Hazel A, Bathen M, Mozaffar T. PMID: 22246880; PMCID: PMC3262776.
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Valosin-containing protein mutation and Parkinson's disease. Parkinsonism Relat Disord. 2012 Jan; 18(1):107-9. Chan N, Le C, Shieh P, Mozaffar T, Khare M, Bronstein J, Kimonis V. PMID: 21816654.
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Phase II screening trial of lithium carbonate in amyotrophic lateral sclerosis: examining a more efficient trial design. Neurology. 2011 Sep 06; 77(10):973-9. Miller RG, Moore DH, Forshew DA, Katz JS, Barohn RJ, Valan M, Bromberg MB, Goslin KL, Graves MC, McCluskey LF, McVey AL, Mozaffar T, Florence JM, Pestronk A, Ross M, Simpson EP, Appel SH, WALS Study Group. PMID: 21813790; PMCID: PMC3171956.
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Correspondence regarding: TDP-43 proteinopathy and motor neuron disease in chronic traumatic encephalopathy. J Neuropathol Exp Neurol 2010:69;918-29. J Neuropathol Exp Neurol. 2011 Jan; 70(1):96-7; author reply 98-100. Bedlack RS, Genge A, Amato AA, Shaibani A, Jackson CE, Kissel JT, Wall C, King WM, Cupler E, Lou JS, Ensrud E, Tan E, Goldstein JM, Katz J, Dimachkie MM, Barohn RJ, Mozaffar T. PMID: 21173608.
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Congenital myasthenic syndrome associated with epidermolysis bullosa caused by homozygous mutations in PLEC1 and CHRNE. Clin Genet. 2011 Nov; 80(5):444-51. Maselli RA, Arredondo J, Cagney O, Mozaffar T, Skinner S, Yousif S, Davis RR, Gregg JP, Sivak M, Konia TH, Thomas K, Wollmann RL. PMID: 21175599.
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Compressive neuropathies of the upper extremity: update on pathophysiology, classification, and electrodiagnostic findings. J Hand Surg Am. 2010 Apr; 35(4):668-77. Tapadia M, Mozaffar T, Gupta R. PMID: 20223605; PMCID: PMC4715364.
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Clinical findings in MuSK-antibody positive myasthenia gravis: a U.S. experience. Muscle Nerve. 2010 Mar; 41(3):370-4. Pasnoor M, Wolfe GI, Nations S, Trivedi J, Barohn RJ, Herbelin L, McVey A, Dimachkie M, Kissel J, Walsh R, Amato A, Mozaffar T, Hungs M, Chui L, Goldstein J, Novella S, Burns T, Phillips L, Claussen G, Young A, Bertorini T, Oh S. PMID: 19882635.
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Neuromuscular junction integrity after chronic nerve compression injury. J Orthop Res. 2009 Jan; 27(1):114-9. Mozaffar T, Strandberg E, Abe K, Hilgenberg LG, Smith MA, Gupta R. PMID: 18655131; PMCID: PMC2670070.
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A novel, efficient, randomized selection trial comparing combinations of drug therapy for ALS. Amyotroph Lateral Scler. 2008 Aug; 9(4):212-22. 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. PMID: 18608093; PMCID: PMC4354803.
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Myopathy associated with chronic orlistat consumption: a case report. Neuromuscul Disord. 2008 May; 18(5):410-2. Ringman JM, Mozaffar T. PMID: 18430571.
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The spectrum of neurological complications in Pakistani patients with malignancies. J Pak Med Assoc. 2008 Apr; 58(4):160-4. Sharfuddin AA, Fatima H, Jilani SM, Mozaffar FH, Mozaffar T. PMID: 18655420.
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Rapid identification of mitochondrial DNA (mtDNA) mutations in neuromuscular disorders by using surveyor strategy. Mitochondrion. 2008 Mar; 8(2):136-45. Bannwarth S, Procaccio V, Rouzier C, Fragaki K, Poole J, Chabrol B, Desnuelle C, Pouget J, Azulay JP, Attarian S, Pellissier JF, Gargus JJ, Abdenur JE, Mozaffar T, Calvas P, Labauge P, Pages M, Wallace DC, Lambert JC, Paquis-Flucklinger V. PMID: 18078792.
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Efficacy of minocycline in patients with amyotrophic lateral sclerosis: a phase III randomised trial. Lancet Neurol. 2007 Dec; 6(12):1045-53. Gordon PH, Moore DH, Miller RG, Florence JM, Verheijde JL, Doorish C, Hilton JF, Spitalny GM, MacArthur RB, Mitsumoto H, Neville HE, Boylan K, Mozaffar T, Belsh JM, Ravits J, Bedlack RS, Graves MC, McCluskey LF, Barohn RJ, Tandan R, Western ALS Study Group. PMID: 17980667.
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The role of neurodiagnostic studies in nerve injuries and other orthopedic disorders. J Hand Surg Am. 2007 Oct; 32(8):1280-90. Strandberg EJ, Mozaffar T, Gupta R. PMID: 17923316.
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N-glycan processing deficiency promotes spontaneous inflammatory demyelination and neurodegeneration. J Biol Chem. 2007 Nov 16; 282(46):33725-33734. Lee SU, Grigorian A, Pawling J, Chen IJ, Gao G, Mozaffar T, McKerlie C, Demetriou M. PMID: 17855338.
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Whole-genome analysis of sporadic amyotrophic lateral sclerosis. N Engl J Med. 2007 Aug 23; 357(8):775-88. Dunckley T, Huentelman MJ, Craig DW, Pearson JV, Szelinger S, Joshipura K, Halperin RF, Stamper C, Jensen KR, Letizia D, Hesterlee SE, Pestronk A, Levine T, Bertorini T, Graves MC, Mozaffar T, Jackson CE, Bosch P, McVey A, Dick A, Barohn R, Lomen-Hoerth C, Rosenfeld J, O'connor DT, Zhang K, Crook R, Ryberg H, Hutton M, Katz J, Simpson EP, Mitsumoto H, Bowser R, Miller RG, Appel SH, Stephan DA. PMID: 17671248.
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Molecular and cellular defects of skeletal muscle in an animal model of acute quadriplegic myopathy. Muscle Nerve. 2007 Jan; 35(1):55-65. Mozaffar T, Haddad F, Zeng M, Zhang LY, Adams GR, Baldwin KM. PMID: 16967495.
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Minocycline-induced skin and dental pigmentations. Neurology. 2006 Dec 26; 67(12):2185. Mozaffar T, Gordon PH. PMID: 17190941.
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Delayed oxaliplatin-associated neurotoxicity following adjuvant chemotherapy for stage III colon cancer. Anticancer Drugs. 2006 Jan; 17(1):103-5. Choi J, Kong K, Mozaffar T, Holcombe RF. PMID: 16317297.
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Schwann cells upregulate vascular endothelial growth factor secondary to chronic nerve compression injury. Muscle Nerve. 2005 Apr; 31(4):452-60. Gupta R, Gray M, Chao T, Bear D, Modafferi E, Mozaffar T. PMID: 15685607.
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Critical care myopathy: an emerging medical catastrophe. J Pak Med Assoc. 2003 Dec; 53(12):608-11. Mozaffar T, Mozaffar FH. PMID: 14765943.
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Ischemic stroke subtypes in Pakistan: the Aga Khan University Stroke Data Bank. J Pak Med Assoc. 2003 Dec; 53(12):584-8. Syed NA, Khealani BA, Ali S, Hasan A, Akhtar N, Brohi H, Mozaffar T, Ahmed N, Hameed A, Baig SM, Wasay M. PMID: 14765937.
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Macrophage recruitment follows the pattern of inducible nitric oxide synthase expression in a model for carpal tunnel syndrome. J Neurotrauma. 2003 Jul; 20(7):671-80. Gupta R, Lin YM, Bui P, Chao T, Preston C, Mozaffar T. PMID: 12908928.
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PIP2 binding residues of Kir2.1 are common targets of mutations causing Andersen syndrome. Neurology. 2003 Jun 10; 60(11):1811-6. Donaldson MR, Jensen JL, Tristani-Firouzi M, Tawil R, Bendahhou S, Suarez WA, Cobo AM, Poza JJ, Behr E, Wagstaff J, Szepetowski P, Pereira S, Mozaffar T, Escolar DM, Fu YH, Ptácek LJ. PMID: 12796536.
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Predictors of mortality in brain abscess. J Pak Med Assoc. 2002 Mar; 52(3):111-6. Qureshi HU, Habib AA, Siddiqui AA, Mozaffar T, Sarwari AR. PMID: 12071065.
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The neuropathology of leprosy. Arch Neurol. 2002 Jan; 59(1):138-40. Habib AA, Mozaffar T. PMID: 11790242.
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Brain abscess. Arch Neurol. 2001 Aug; 58(8):1302-4. Habib AA, Mozaffar T. PMID: 11493176.
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Critical illness myopathy. Muscle Nerve. 2001 Jul; 24(7):973-4. Mozaffar T. PMID: 11410928.
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Cerebral artery air embolism following an esophagogastroscopy: a case report. Neurology. 2001 Jan 09; 56(1):136-7. Akhtar N, Jafri W, Mozaffar T. PMID: 11148258.
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Gram negative meningitis resulting from rupture of a spinal epidural abscess into the subarachnoid space. J Pak Med Assoc. 2000 Nov; 50(11):393-4. Lodhi S, Mozaffar T, Sarwari AR. PMID: 11126818.
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Factor XI deficiency-related spontaneous primary intraventricular hemorrhage. South Med J. 2000 Oct; 93(10):1017-8. Khealani B, Farhat Z, Mozaffar T. PMID: 11147466.
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Myopathy with anti-Jo-1 antibodies: pathology in perimysium and neighbouring muscle fibres. J Neurol Neurosurg Psychiatry. 2000 Apr; 68(4):472-8. Mozaffar T, Pestronk A. PMID: 10727483; PMCID: PMC1736872.
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Chronic subdural haematoma presenting with transient ischaemic attacks--a case report. Ann Acad Med Singap. 1999 Nov; 28(6):861-2. Khealani B, Mozaffar T. PMID: 10672404.
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Dysphagia in hereditary sensory autonomic neuropathy type IV. J Pak Med Assoc. 1999 May; 49(5):121-3. Shah U, Arshad M, Mozaffar T. PMID: 10555429.
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Neuralgic amyotrophy (Parsonage-Turner syndrome): an often misdiagnosed diagnosis. J Pak Med Assoc. 1999 Apr; 49(4):101-3. Saleem F, Mozaffar T. PMID: 10540541.
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Clinical correlates of granulomas in muscle. J Neurol. 1998 Aug; 245(8):519-24. Mozaffar T, Lopate G, Pestronk A. PMID: 9747915.
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Paraneoplastic necrotizing myopathy: clinical and pathological features. Neurology. 1998 Mar; 50(3):764-7. Levin MI, Mozaffar T, Al-Lozi MT, Pestronk A. PMID: 9521271.
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Case 21-1997: Paraneoplastic cerebellar degeneration and Hodgkin's disease. N Engl J Med. 1998 Jan 01; 338(1):66. Mozaffar T. PMID: 9424575.
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