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T. Mozaffar · SciRah

Researcher profile · Semantic Scholar

T. Mozaffar

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T. Mozaffar — researcher profile on SciRah

h-index: 50
Works: 367
Citations: 8,875

Most-cited papers

Safety and Efficacy of Autologous RNA Chimeric Antigen Receptor T-cell (rCAR-T) Therapy in Myasthenia Gravis: a prospective, multicenter, open-label, non-randomised phase 1b/2a study
V. Granit, M. Benatar, M. Kurtoğlu, M. Miljkovic +10 more
2023Lancet Neurology183 citations
Semantic Scholar
Single-cell and spatial transcriptomics identify a macrophage population associated with skeletal muscle fibrosis
G. Coulis, D. Jaime, C. Guerrero-Juarez, J. Kastenschmidt +31 more
2023Science Advances103 citations Open access
Semantic Scholar
The Miami Framework for ALS and related neurodegenerative disorders: an integrated view of phenotype and biology
M. Benatar, J. Wuu, E. Huey, C. McMillan +49 more
2024Nature Reviews Neurology52 citations Open access
Semantic Scholar
Case study of CD19 CAR T therapy in a subject with immune-mediate necrotizing myopathy treated in the RESET-Myositis phase I/II trial
J. Volkov, Daniel Nunez, T. Mozaffar, J. Stadanlick +14 more
2024Molecular Therapy51 citations Open access
Semantic Scholar
Efficacy and Safety of Avalglucosidase Alfa in Patients With Late-Onset Pompe Disease After 97 Weeks
P. Kishnani, J. Diaz-Manera, A. Toscano, P. Clemens +23 more
2023JAMA Neurology41 citations Open access
Semantic Scholar
Safety and outcomes with efgartigimod use for acetylcholine receptor‐positive generalized myasthenia gravis in clinical practice
N. Katyal, Karen Halldorsdottir, Raghav Govindarajan, Perry Shieh +11 more
2023Muscle and Nerve36 citations
Semantic Scholar
BCMA-directed mRNA CAR T cell therapy for myasthenia gravis: a randomized, double-blind, placebo-controlled phase 2b trial
T. Vu, H. Durmuş, M. Rivner, S. Shroff +26 more
2026Nature Medicine30 citations Open access
Semantic Scholar
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)
B. Schoser, Priya S. Kishnani, D. Bratkovic, Barry J. Byrne +79 more
2024Journal of Neurology28 citations Open access
Semantic Scholar
Safety and efficacy of arimoclomol for inclusion body myositis: a multicentre, randomised, double-blind, placebo-controlled trial.
P. Machado, Michael P. McDermott, T. Blaettler, Claus Sundgreen +20 more
2023Lancet Neurology28 citations Open access
Semantic Scholar
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)
Barry J. Byrne, B. Schoser, Priya S. Kishnani, D. Bratkovic +16 more
2023Journal of Neurology22 citations Open access
Semantic Scholar
BCMA-directed mRNA CAR-T cell therapy for myasthenia gravis: exploratory biomarker analysis of a placebo-controlled phase 2b trial
Renee R. Fedak, Rachel N Ruggerie, Yufei Shan, Elizabeth J. Curvino +65 more
2026Nature Medicine21 citations Open access
Semantic Scholar
Single-cell and spatial transcriptomics identify a macrophage population associated with skeletal muscle fibrosis
G. Coulis, D. Jaime, C. Guerrero-Juarez, J. Kastenschmidt +30 more
2023bioRxiv21 citations Open access
Semantic Scholar
Durability of Response to B‐Cell Maturation Antigen‐Directed mRNA Cell Therapy in Myasthenia Gravis
N. Chahin, G. Sahagian, M. Feinberg, C. A. Stewart +6 more
2025Annals of Clinical and Translational Neurology16 citations Open access
Semantic Scholar
Cipaglucosidase alfa plus miglustat: linking mechanism of action to clinical outcomes in late-onset Pompe disease
Barry J. Byrne, G. Parenti, B. Schoser, A. T. van der Ploeg +13 more
2024Frontiers in Neurology16 citations Open access
Semantic Scholar
Safety and efficacy of chronic weekly rozanolixizumab in generalized myasthenia gravis: the randomized open-label extension MG0004 study
V. Bril, A. Drużdż, J. Grosskreutz, A. Habib +135 more
2025Journal of Neurology11 citations Open access
Semantic Scholar
Efficacy and safety of maintenance intravenous immunoglobulin in generalized myasthenia gravis patients with acetylcholine receptor antibodies: A multicenter, double‐blind, placebo‐controlled trial
V. Bril, Tomasz Berkowicz, Andrzej Szczudlik, Michael W. Nicolle +21 more
2024Muscle and Nerve11 citations Open access
Semantic Scholar
Efficacy and safety of avalglucosidase alfa in patients with late-onset Pompe disease after 145 weeks of treatment during the COMET trial
Priya S. Kishnani, J. Diaz-Manera, S. Illarioshkin, A. T. van der Ploeg +21 more
2025Journal of Neurology10 citations Open access
Semantic Scholar
Switching treatment to cipaglucosidase alfa plus miglustat positively affects patient-reported outcome measures in patients with late-onset Pompe disease
Priya S. Kishnani, Barry J. Byrne, K. Claeys, J. Diaz-Manera +73 more
2024Journal of Patient-Reported Outcomes10 citations Open access
Semantic Scholar
Safety and Tolerability of BCMA-Directed mRNA CAR T-Cell Therapy in Multiple Myeloma and Autoimmune Disease
Milos D. Miljkovic, A. Asch, Gregory Orloff, R. Boccia +10 more
2024Blood9 citations
Semantic Scholar
Provisional practice recommendation for the management of myopathy in VCP‐associated multisystem proteinopathy
B. Roy, A. Peck, T. Evangelista, G. Pfeffer +16 more
2023Annals of Clinical and Translational Neurology9 citations Open access
Semantic Scholar
Twelve-Month Follow-Up of Patients With Generalized Myasthenia Gravis Receiving BCMA-Directed mRNA Cell Therapy
N. Chahin, G. Sahagian, M. Feinberg, C. A. Stewart +6 more
2024medRxiv8 citations Open access
Semantic Scholar
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
S. Shohet, Noemi Hummel, Shuai Fu, Ian Keyzor +6 more
2024Journal of Comparative Effectiveness Research8 citations Open access
Semantic Scholar
A Phase 3 Trial of Brepocitinib in Dermatomyositis.
R. Vleugels, Julie J. Paik, I. Bauer Ventura, Aaron R. Mangold +37 more
2026New England Journal of Medicine7 citations Open access
Semantic Scholar
Switching Enzyme Replacement Therapy for Late‐Onset Pompe Disease From Alglucosidase Alfa to Cipaglucosidase Alfa Plus Miglustat: Post Hoc Effect Size Analysis of PROPEL
Hani Kushlaf, J. Díaz-Manera, D. Bratkovic, Barry J. Byrne +13 more
2025Muscle and Nerve7 citations Open access
Semantic Scholar
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
A. Peck, A. Dadi, Z. Yavarow, L. Alfano +37 more
2025Neurobiology of Disease7 citations Open access
Semantic Scholar
Consensus nomenclature and abbreviation for anti-synthetase syndrome: an IMACS project.
Anushka Aggarwal, Tanya Chandra, S. Keret, J. Pauling +32 more
2025Rheumatology6 citations Open access
Semantic Scholar
Prospective observational study of FKRP‐related limb‐girdle muscular dystrophy R9: A GRASP consortium study
L. Alfano, Meredith K. James, Kristine Grosfjeld Petersen, Karen S. Rudolf +27 more
2024Annals of Clinical and Translational Neurology6 citations Open access
Semantic Scholar
Defining clinical endpoints in limb girdle muscular dystrophy: a GRASP-LGMD study
Amy Doody, L. Alfano, J. Diaz-Manera, L. Lowes +14 more
2024BMC Neurology6 citations Open access
Semantic Scholar
Change in Concomitant Therapies for Generalized Myasthenia Gravis in Patients Receiving Eculizumab: a Retrospective Analysis of Registry Data (P1-5.002)
M. Pulley, S. Macwan, R. Nowak, T. Mozaffar +4 more
2023Sunday, April 236 citations
Semantic Scholar
Efficacy and safety of avalglucosidase alfa in participants with late-onset Pompe disease after 145 weeks of treatment during the COMET trial
P. Kishnani, J. Diaz-Manera, H. Kushlaf, Shafeeq Ladha +20 more
2023Molecular Genetics and Metabolism5 citations
Semantic Scholar
The Mythology of Polymyositis
Suur Biliciler, T. Mozaffar
2025Rheumatic Disease Clinics of North America4 citations Open access
Semantic Scholar
Clinical Trial Readiness in Limb Girdle Muscular Dystrophy R1 (LGMDR1): A GRASP Consortium Study
Stephanie M. Hunn, L. Alfano, Aileen S. Jones, Amanda Butler +25 more
2025Annals of Clinical and Translational Neurology4 citations Open access
Semantic Scholar
Debamestrocel multimodal effects on biomarker pathways in amyotrophic lateral sclerosis are linked to clinical outcomes
Stacy R. Lindborg, Namita A. Goyal, Jonathan Katz, Matthew J Burford +23 more
2024Muscle and Nerve4 citations Open access
Semantic Scholar
Editorial: Inflammatory muscle diseases: an update
J. Tanboon, M. Needham, T. Mozaffar, W. Stenzel +1 more
2023Frontiers in Neurology4 citations Open access
Semantic Scholar
Effects of enzyme replacement therapy on bone density in late onset Pompe disease.
M. Avanti, Angela Martin, R. C. Columbres, T. Mozaffar +1 more
2023Molecular Genetics and Metabolism3 citations Open access
Semantic Scholar
Standardized Interoperable Data Collection for Myositis Research: Developing Expert Consensus on Common Data Elements for Myositis Outcome Measures
D. Saygin, Matthew Diller, Varsha Surampudi, M. Bodkin +32 more
2026Arthritis care & research2 citations Open access
Semantic Scholar
Efficacy and Safety of Autologous BCMA-directed mRNA CAR T-Cell Therapy in Generalized Myasthenia Gravis: Results from a Phase 2b Randomized Placebo-controlled Trial (S34.002)
Tuan Vu, H. D. Tekce, M. Rivner, S. Shroff +17 more
2025Neurology2 citations
Semantic Scholar
Aging Accelerates Degradation of Human Neuromuscular Junction Following Peripheral Nerve Injury
Luigi P. Gonzales, Vivian Y. Chen, Amanda Tedesco, Saman Andalib +6 more
2025Muscle and Nerve2 citations
Semantic Scholar
Recurring homozygous ACTN2 variant (p.Arg506Gly) causes a recessive myopathy
S. Donkervoort, P. Mohassel, M. O'Leary, D. Bonner +19 more
2024Annals of Clinical and Translational Neurology2 citations Open access
Semantic Scholar
Treatment Fluctuations in Patients with Acetylcholine Receptor Antibody Positive Generalized Myasthenia Gravis on Maintenance Ravulizumab Treatment (P10-11.001)
D. Ho, Katherine Clifford, Samantha Blum, M. Freimer +8 more
2024Neurology2 citations
Semantic Scholar
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
T. Mozaffar, Lionel Riou França, J. Msihid, Pragya Shukla +6 more
2024Molecular Genetics and Metabolism Reports2 citations Open access
Semantic Scholar
Indirect treatment comparison of three enzyme replacement treatments for late-onset Pompe disease: A network meta-analysis with patient-level and aggregate data
J. Castelli, Shuai Fu, N. Hummel, T. Mozaffar +5 more
2023Molecular Genetics and Metabolism2 citations
Semantic Scholar
Quantitative Measurement of Glycosylated ⍺-Dystroglycan as a Biomarker for Disease Severity in Limb-Girdle Muscular Dystrophy Type 2I/R9
J. Vissing, T. Mozaffar, Nicholas E. Johnson, Kathy Mathews +20 more
2026Neurology: Genetics1 citations
Semantic Scholar
208-week outcomes of cipaglucosidase alfa plus miglustat in patients with late-onset Pompe disease treated from PROPEL baseline: Pulmonary function
Priya S. Kishnani, D. Bratkovic, K. Claeys, J. Diaz-Manera +9 more
2026Molecular Genetics and Metabolism1 citations
Semantic Scholar
208-week outcomes of cipaglucosidase alfa plus miglustat in patients with late-onset Pompe disease treated from PROPEL baseline: Muscle function and biomarkers
T. Mozaffar, D. Bratkovic, K. Claeys, J. Diaz-Manera +8 more
2026Molecular Genetics and Metabolism1 citations Open access
Semantic Scholar
Myositis in 2025: Defining Subtypes, Closing Gaps, and Shaping the Future.
Julie J. Paik, T. Mozaffar
2025Rheumatic Disease Clinics of North America1 citations
Semantic Scholar
Azt845 gene replacement therapy for late-onset Pompe disease: An update on safety and preliminary efficacy data from FORTIS, a phase 1/2 open-label clinical study
T. Mozaffar, N. Longo, Mark Walzer, Achim Steup +3 more
2025Molecular Genetics and Metabolism1 citations
Semantic Scholar
The Dermatomyositis Disease Symptom Questionnaire (DM-DSQ): A Measure to Assess the Patient Experience of Dermatomyositis Symptoms
L. Christopher-Stine, A. Ciesluk, H. Chinoy, Namita A. Goyal +8 more
2024Journal of Rheumatology1 citations Open access
Semantic Scholar
Chimeric Antigen Receptor (CAR) T-cell Therapy in Neurological Disorders
Ali A. Habib, S. Ciurea, Hafsa I. Kamboh, M. Miljković +1 more
2024Neurology1 citations
Semantic Scholar
Effect size analysis of cipaglucosidase alfa plus miglustat versus alglucosidase alfa in ERT-experienced adults with late-onset Pompe disease in PROPEL
T. Mozaffar, D. Bratkovic, B. Byrne, K. Claeys +11 more
2024Molecular Genetics and Metabolism1 citations Open access
Semantic Scholar

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J. Diaz-Manera×11
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B. Schoser×10
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M. Dimachkie×9
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Priya S. Kishnani×8
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