Professor, Neurology
School of Medicine

Professor, Orthopaedic Surgery
School of Medicine

Chair, Institutional Review Board A, Research Administration

Director, Division of Neuromuscular Diseases, Neurology
School of Medicine

Director, UC Irvine-MDA ALS and Neuromuscular Center, Neurology
School of Medicine

Affiliate Faculty, Institute for Memory Impairments and Neurological Disorders (UCI MIND)

Professor, Pathology
School of Medicine

Mechanisms underlying muscle atrophy in various neuromuscular disorders; Inclusion Body Myositis; Natural History of Rare and Ultra Rare Myopathies; ; Clinical Trials

www.ucihealth.com/als

Board Certification in Neurology, 1996-2026
Sven Elliason Faculty Award for Teaching Excellence, Department of Neurology, Washington University in St. Louis, 1997
Faculty Teaching Award, Department of Neurology University of California, Irvine, 2001, 2002, 2003, 2007
National Teaching Recognition, American Academy of Neurology, 2003
Elected Fellow, American Academy of Neurology, 2007
Elected member American Neurological Association, 2007
President, Orange County Neurological Society, 2006-2008
Board Certification in Neuromuscular Medicine, 2008-2028
Board Certification in Clinical Neuromuscular Pathology, 2015 - 2025

I am the Principal and the coordinating Investigator for a multi-center natural history study in sporadic Inclusion Body Myositis (sIBM) that is currently being considered for funding by NIH. sIBM is a rare disorder of aging Americans, causing asymmetric muscle weakness and severe disability and morbidity. It is currently untreatable, and poorly understood. The prevalence of sIBM is likely to increase as the proportion of the United States population above the age of 65 years continues to grow. A major barrier to clinical trials in sIBM has been the lack of full understanding of the natural history of the disease. It remains to be determined whether the rates of disease progression is uniform and whether the various biomarkers associated with sIBM (anti-NT5c1A antibodies, variant T-cell populations) influence the natural history and disease behavior. Given the slow rate of disease progression, such observations cannot be made in the context of a routine clinical trial, and such studies need to be done as a separate stand-alone observational study. To address these unmet needs, we propose a prospective study with four specific aims. Aim 1: To determine for the first time whether c1A antibodies mediate disease progression over a two year interval in patients with sIBM. Aim 2: To perform a detailed morphological, histochemical, and immunohistochemical analysis of fresh muscle biopsy specimens obtained from a subset of patients with sIBM. Aim 3: To characterize the distribution of “immunosenescent” lymphocytes in circulating blood from patients with sIBM. Aim 4: To quantify the decline in the respiratory function of sIBM patients. The significance of our proposed study is 1) to allow for a detailed characterization of the disease progression in sIBM over a two-year period, and 2) to explore the relationship of a number of biomarkers associated with sIBM, and their influence on disease behavior and disease progression. Upon completion of these aims, we will 1) understand the disease phenotype, including pattern of respiratory involvement, and disease progression in sIBM better and understand the influence of serum antibodies to NT5c1A antibodies on the natural history and disease behavior; 2) define differences in serum variant T-cells and cytokine signatures in sIBM patients and their influence on disease progression and behavior; and 3) understand muscle pathology and immune cell distribution in sIBM patients and its relationship to NT5c1A antibodies. These findings may influence future trial design in sIBM. Finally, we will have created a thirteen-site consortium of myositis treatment centers that will be ready to adopt quickly any future clinical trials aimed at changing the course of sIBM.

2017 – 2020 Dr. Stanley van den Noort Endowed Chair in Neurology
2015 - 2020 Elected, Best Doctors, Best Doctors, Inc
2008, 2010-2020 Elected, Physician of Excellence, Orange County Medical Association
2012-2013, 2017-2020 Listed in Top Doctors, US News in collaboration with Castle-Connolly

Dr. Tahseen Mozaffar is a Professor of Neurology, Orthopaedic Surgery and Pathology and Laboratory Medicine at University of California, Irvine. He is the Director of the UC Irvine-MDA ALS and Neuromuscular Center and the Director of the Division of Neuromuscular Diseases. Dr. Mozaffar serves as chair of one of the biomedical committees and the institutional liaison for Trials Innovation Hub for the Center for Translational Sciences Award (CTSA) at University of California, Irvine. He is the Principal Investigator for UCI-NEXT, the NeuroNEXT award to the University of California, Irvine, one of 25 such NeuroNEXT sites funded by the NINDS/NIH.

He graduated medical school at the Aga Khan University in Karachi, Pakistan in 1989. After a rotating internship in Internal Medicine and General Surgery at the Aga Khan University Hospital, he came to the US for neurology residency training. He completed neurology residency training at Barnes Hospital in St. Louis, Missouri in 1995 and then completed a two clinical and research fellowship in Neuromuscular Disorders at Washington University in 1997. After a three-year faculty appointment at the Aga Khan University in Karachi, Pakistan, Dr. Mozaffar returned to the US and was appointed as Assistant Professor of Neurology and Director, UC Irvine Neuromuscular Program University of California, Irvine in 2000, where he has built a nationally recognized clinical and research program in Neuromuscular Disorders.

He is actively involved in clinical and translational research in Neuromuscular Disorders, including currently serving as Principal Site Investigator on over a dozen clinical trials in myasthenia gravis, rare and ultra-rare myopathies and in immune myopathies. He has co-authored over 150 peer-reviewed publications and has authored or co-authored over a dozen book chapters and invited reviews. As an expert in these rare and ultra-rare myopathies, he is actively sought as an advisor by pharmaceutical companies for trial design and identifying disease targets. He serves on a global advisory board for Pompe at Amicus and for Spark Therapeutics. He serves on the Data Safety Monitoring Board for Acceleron. He is a standing reviewer for the ETTN study section at the NIH.

He is a member of the Medical Advisory Board for the Myositis Association and recently finished his term on the executive committee of the Muscle Study Group. He served on the conference planning committee for the MDA Clinic Directors Conference, 2nd Global conference on myositis (GCOM), the 3rd Global Myositis Conference, the Muscle Study Group, and the International Congress on Neuromuscular Disorders. He is the Director of the nationally recognized Annual UC Irvine Neuromuscular Colloquia, now in its 10th year of existence and Director of the Annual Neuromuscular Pathology Colloquium, now in its 5th year.

Dr. Mozaffar’s immunological research interests include characterization of the myopathology in various forms of immune myopathies, including the first description of myopathology in Jo-1 antibody associated myositis. Based on his seminal work, this form of immune myopathy has now been given a separate classification of anti-synthetase syndrome. His most recent work has been in various subtypes of dermatomyositis, immune mediated necrotizing myopathy and in collaboration with Professor S. Armando Villalta, a deep phenotyping of the immune infiltrates in sporadic inclusion body myositis. His other area of interest is myasthenia gravis, an autoimmune disease of the muscle. He is the site PI for the first CAR-T cell therapy trial in MG in collaboration with Cartesian Inc.

Access my bibliography (over 160 peer-reviewed journal articles) using this link:

www.ncbi.nlm.nih.gov/sites/myncbi/tahseen.mozaffar.1/bibliography/49655508/public/?sort=date&direction=ascending

NS107210: UCI-NEXT, a NeuroNEXT site

1R01AR078340-01: Influence of NT5c1A antibodies on disease progression, clinical phenotype and blood and muscle biomarkers in sporadic Inclusion Body Myositis – A prospective evaluation

American Academy of Neurology

World Muscle Society

American Neurological Association

UC Irvine-MDA ALS and Neuromuscular Center

Peripheral Nerve Research Laboratory

Institute for Memory Impairments and Neurological Disorders (UCI MIND)

Institute for Immunology

Link to this profile
https://faculty.uci.edu/profile/?facultyId=4612

Last updated
04/06/2021