TahseenMozaffarMD

PubMed

• Debamestrocel multimodal effects on biomarker pathways in amyotrophic lateral sclerosis are linked to clinical outcomes.
  Stacy R Lindborg, Namita A Goyal, Jonathan Katz, Matthew Burford, Jenny Li, Haggai Kaspi, Natalie Abramov, Bruno Boulanger, James D Berry, Katharine Nicholson, Tahseen...> ;Muscle & Nerve. 2024 Apr 9
• Defining clinical endpoints in limb girdle muscular dystrophy: a GRASP-LGMD study.
  Amy Doody, Lindsay Alfano, Jordi Diaz-Manera, Linda Lowes, Katherine D Mathews, Matthew Wicklund, Man Hung, Nicholas E Johnson, , Tahseen Mozaffar, Conrad C Weihl, Jef...> ;BMC Neurology. 2024 Mar 15
• 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).
  Benedikt Schoser, Priya S Kishnani, Drago Bratkovic, Barry J Byrne, Kristl G Claeys, Jordi Díaz-Manera, Pascal Laforêt, Mark Roberts, Antonio Toscano, Ans T van der Pl...> ;Journal of Neurology. 2024 Feb 28
• Recurring homozygous ACTN2 variant (p.Arg506Gly) causes a recessive myopathy.
  Sandra Donkervoort, Payam Mohassel, Melanie O'Leary, Devon E Bonner, Taila Hartley, Nicole Acquaye, Astrid Brull, Tahseen Mozaffar, Mario A Saporta, David A Dyment, Ja...> ;Annals of Clinical and Translational Neurology. 2024 Mar 1
• 2 citations
  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, Benedikt Schoser, Priya S Kishnani, Drago Bratkovic, Paula R Clemens, Ozlem Goker-Alpan, Xue Ming, Mark Roberts, Matthias Vorgerd, Kumaraswamy Sivakumar...> ;Journal of Neurology. 2024 Apr 1
• Defining Clinical Endpoints in Limb Girdle Muscular Dystrophy: A GRASP-LGMD study.
  Amy Doody, Lindsay Alfano, Jordi Diaz-Manera, Linda Lowes, Tahseen Mozaffar, Kathy Mathews, Conrad C Weihl, Matthew Wicklund, Jeffery Statland, Nicholas E Johnson> ;Research Square. 2023 Oct 6
• 3 citations
  Safety and efficacy of arimoclomol for inclusion body myositis: a multicentre, randomised, double-blind, placebo-controlled trial.
  Pedro M Machado, Michael P McDermott, Thomas Blaettler, Claus Sundgreen, Anthony A Amato, Emma Ciafaloni, Miriam Freimer, Summer B Gibson, Sarah M Jones, Todd D Levine...> ;The Lancet. Neurology. 2023 Oct 1
• 4 citations
  Safety and outcomes with efgartigimod use for acetylcholine receptor-positive generalized myasthenia gravis in clinical practice.
  Nakul Katyal, Karen Halldorsdottir, Raghav Govindarajan, Perry Shieh, Suraj Muley, Phoebedel Reyes, Kenneth K Leung, Jeffrey Mullen, Shadi Milani-Nejad, Manisha Korb, ...> ;Muscle & Nerve. 2023 Nov 1
• 8 citations
  Single-cell and spatial transcriptomics identify a macrophage population associated with skeletal muscle fibrosis.
  Gerald Coulis, Diego Jaime, Christian Guerrero-Juarez, Jenna M Kastenschmidt, Philip K Farahat, Quy Nguyen, Nicholas Pervolarakis, Katherine McLinden, Lauren Thurlow, ...> ;Science Advances. 2023 Jul 7
• 15 citations
  Safety and clinical activity of autologous RNA chimeric antigen receptor T-cell therapy in myasthenia gravis (MG-001): a prospective, multicentre, open-label, non-rand...
  Volkan Granit, Michael Benatar, Metin Kurtoglu, Miloš D Miljković, Nizar Chahin, Gregory Sahagian, Marc H Feinberg, Adam Slansky, Tuan Vu, Christopher M Jewell, Michae...> ;The Lancet. Neurology. 2023 Jul 1
• Effects of enzyme replacement therapy on bone density in late onset Pompe disease.
  Mahima Avanti, Angela Martin, Rod Carlo Columbres, Tahseen Mozaffar, Virginia Kimonis> ;Molecular Genetics and Metabolism. 2023 Nov 1
• 1 citations
  Single-cell and spatial transcriptomics identify a macrophage population associated with skeletal muscle fibrosis.
  Gerald Coulis, Diego Jaime, Christian Guerrero-Juarez, Jenna M Kastenschmidt, Philip K Farahat, Quy Nguyen, Nicholas Pervolarakis, Katherine McLinden, Lauren Thurlow, ...> ;Biorxiv. 2023 Apr 18
• 7 citations
  Efficacy and Safety of Avalglucosidase Alfa in Patients With Late-Onset Pompe Disease After 97 Weeks: A Phase 3 Randomized Clinical Trial.
  Priya S Kishnani, Jordi Diaz-Manera, Antonio Toscano, Paula R Clemens, Shafeeq Ladha, Kenneth I Berger, Hani Kushlaf, Volker Straub, Gerson Carvalho, Tahseen Mozaffar,...> ;JAMA Neurology. 2023 Jun 1
• 3 citations
  Provisional practice recommendation for the management of myopathy in VCP-associated multisystem proteinopathy.
  Bhaskar Roy, Allison Peck, Teresinha Evangelista, Gerald Pfeffer, Leo Wang, Jordi Diaz-Manera, Manisha Korb, Matthew P Wicklund, Margherita Milone, Miriam Freimer, Han...> ;Annals of Clinical and Translational Neurology. 2023 May 1
• 1 citations
  Imaging beyond muscle magnetic resonance imaging in inclusion body myositis.
  Namita A Goyal, Tahseen Mozaffar, Mazen M Dimachkie> ;Clinical and Experimental Rheumatology. 2023 Mar 1
• Editorial: Inflammatory muscle diseases: an update.
  Jantima Tanboon, Merrilee Needham, Tahseen Mozaffar, Werner Stenzel, Ichizo Nishino> ;Frontiers in Neurology. 2023 Jan 1
• 9 citations
  Pompe Disease: a Clinical, Diagnostic, and Therapeutic Overview.
  David Stevens, Shadi Milani-Nejad, Tahseen Mozaffar> ;Current Treatment Options in Neurology. 2022 Nov 1
• 4 citations
  Randomized Double-Blind Placebo-Controlled Trial of the Corticosteroid-Sparing Effects of Immunoglobulin in Myasthenia Gravis.
  Vera Bril, Andrzej Szczudlik, Antanas Vaitkus, Csilla Rozsa, Anna Kostera-Pruszczyk, Petr Hon, Josef Bednarik, Michaela Tyblova, Wolfgang Köhler, Toomas Toomsoo, Richa...> ;Neurology. 2023 Feb 14
• 2 citations
  Inclusion body myositis: evolving concepts.
  Mari Perez-Rosendahl, Tahseen Mozaffar> ;Current Opinion in Neurology. 2022 Oct 1
• Facial Onset Sensory and Motor Neuronopathy-Like Syndrome: A Case Report.
  David M Bruss, Prashanth Venkataraman, Tahseen Mozaffar> ;Journal of Clinical Neuromuscular Disease. 2022 Sep 1
• 3 citations
  Correlations of disease severity outcome measures in inclusion body myositis.
  Namita A Goyal, Steven A Greenberg, Jonathan Cauchi, Nadia Araujo, Vivian Li, Marie Wencel, Tyler Irani, Leo H Wang, Anton M Palma, S Armando Villalta, Tahseen Mozaffar> ;Neuromuscular Disorders. 2022 Oct 1
• 17 citations
  Immunophenotyping of Inclusion Body Myositis Blood T and NK Cells.
  Namita A Goyal, Gérald Coulis, Jorge Duarte, Philip K Farahat, Ali H Mannaa, Jonathan Cauchii, Tyler Irani, Nadia Araujo, Leo Wang, Marie Wencel, Vivian Li, Lishi Zhan...> ;Neurology. 2022 Mar 29
• 35 citations
  A randomized placebo-controlled phase 3 study of mesenchymal stem cells induced to secrete high levels of neurotrophic factors in amyotrophic lateral sclerosis.
  Merit E Cudkowicz, Stacy R Lindborg, Namita A Goyal, Robert G Miller, Matthew J Burford, James D Berry, Katharine A Nicholson, Tahseen Mozaffar, Jonathan S Katz, Liber...> ;Muscle & Nerve. 2022 Mar 1
• 1 citations
  COVID-19 infection in patients with late-onset Pompe disease.
  Jennifer Avelar, Marie Wencel, Anastasia Chumakova, Tahseen Mozaffar> ;Muscle & Nerve. 2022 Mar 1
• Utility of video-fundoscopy and prospects of portable stereo-photography of the ocular fundus in neurological patients.
  Tigran Khachatryan, Tahseen Mozaffar, Lilit Mnatsakanyan> ;BMC Neurology. 2022 Feb 19
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Journal Articles

• 224th ENMC International Workshop: Clinico-sero-pathological classification of immune-mediated necrotizing myopathies Zandvoort, The Netherlands, 14-16 October 2016  
  Allenbach Y, Mammen AL, Benveniste O, Stenzel W; Immune-Mediated Necrotizing Myopathies Working Group, Neuromuscul Disord, 1/1/2018
• Randomized study of adjunctive belimumab in participants with generalized myasthenia gravis  
  Hewett K, Sanders DB, Grove RA, Broderick CL, Rudo TJ, Bassiri A, Zvartau-Hind M, Bril V; BEL115123 Study Group, Neurology, 1/1/2018
• A cross-sectional analysis of clinical evaluation in35 individuals with mutations of the valosin-containing protein gene  
  Plewa J, Surampalli A, Wencel M, Milad M, Donkervoort S, Caiozzo VJ, Goyal N, Mozaffar T, Kimonis V, Neuromuscul Disord, 1/1/2018
• Rasagiline for Amyotrophic Lateral Sclerosis: a Randomized Controlled Trial  
  Statland JM, Moore D, Wang Y, Walsh M, Mozaffar T, Elman L, Nations S, Mitsumoto H, Fernandes JA, Saperstein D, Hayat G, Herbelin L, Karam C, Katz J, Wilkins HM, Agbas..., Muscle Nerve, 1/1/2018
• Safety, tolerability, and preliminary efficacy of an IGF-1 mimetic in patients with spinal and bulbar muscular atrophy: a randomized, placebo-controlled trial  
  Grunseich C, Miller R, Swan T, Glass DJ, Mouelhi ME, Fornaro M, Petricoul O, Vostiar I, Roubenoff R, Meriggioli MN, Kokkinis A, Guber RD, Budron MS, Vissing J, Soraru ..., Lancet Neurol, 1/1/2018
• Genotype‐Phenotype Study in Patients with Valosin‐Containing Protein Mutations Associated with Multisystem Proteinopathy  
  V Kimonis, T Mozaffar, C Smith, A K Wang, Clinical Genetics
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  Kishnani P, Tarnopolsky M, Sivakumar K, Roberts M, Dasouki M, Dimachkie M, Finanger E, Goker-Alpan O, Guter K, Mozaffar T, Pervaiz MA, Laforet P, Levine T, Adera M, La..., Molecular Therapy, 1/1/2017
• ALSUntangled No 36: Accilion  
  Bedlack R, Fixsen E, Quinn C, et al., Amyotroph Lateral Scler Frontotemporal Degener, 1/1/2017
• ALSUntangled 38: L-serine  
  Peschansky V, Bedlack R, Quinn C, et al., Amyotroph Lateral Scler Frontotemporal Degener, 1/1/2017
• Genotype-phenotype study in patients with valosin-containing protein mutations associated with multisystem proteinopathy  
  Al-Obeidi E, Al-Tahan S, Surampalli A, Goyal N, Wang A, Hermann A, Omizu M, Smith C, Mozaffar T, Kimonis V, Clin Genet, 1/1/2017
• Defining SOD1 ALS natural history to guide therapeutic clinical trial design  
  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, Zinma..., J Neurol Neurosurg Psychiatry, 1/1/2017
• Sporadic inclusion body myositis misdiagnosed as idiopathic granulomatous myositis  
  Lavian M, Goyal NA, Mozaffar T, Neuromuscul Disord, 1/1/2016
• Safety and efficacy of eculizumab in anti-acetylcholine receptor antibody-positive refractory generalised myasthenia gravis (REGAIN): a phase 3, randomised, double-bli...  
  Howard JF Jr, Utsugisawa K, Benatar M, Murai H, Barohn RJ, Illa I, Jacob S, Vissing J, Burns TM, Kissel JT, Muppidi S, Nowak RJ, O'Brien F, Wang JJ, Mantegazza R; REGA..., Lancet Neurol, 1/1/2016
• Mexilitene for Muscle Cramps in ALS: A Randomized Double-Blind Crossover Trial  
  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, Muscle Nerve, 1/1/2016
• Critically re-evaluating a common technique: Accuracy, reliability and confirmation bias of EMG Response to Charles JA and Souayah N  
  Narayanaswami P, Geisbush T, Jones L, Weiss M, Mozaffar T, Gronseth G, Rutkove, SB, Neurology, 1/1/2016
• ALSUntangled No 31: Protandim  
  Bedlack R, Quinn C, Karam C, et al., Amyotroph Lateral Scler Frontotemporal Degener, 1/1/2016
• ALSUntangled No 32: Gluten-free diet  
  Bedlack R, Quinn C, Karam C, et al., Amyotroph Lateral Scler Frontotemporal Degener, 1/1/2016
• Pulmonary function tests (maximal inspiratory pressure, maximum expiratory pressure, vital capacity, forced vital capacity) predict ventilator and wheelchair use in la...  
  Johnson EM, Roberts M, Mozaffar T, Young P, Quartel A, Berger KI, Neuromuscul Disord, 1/1/2016
• ALS Untangled No33 Endotherapia  
  Bedlack R, Quinn C, Karam C, et al., Amyotroph Lateral Scler Frontotemporal Degener, 1/1/2016
• ALSUntangled No 34: GM604  
  Bedlack R, Quinn C, Karam C, et al., Amyotroph Lateral Scler Frontotemporal Degener, 1/1/2016
• A randomized, placebo-controlled, double-blind phase IIb trial evaluating the safety and efficacy of tirasemtiv in patients with amyotrophic lateral sclerosis  
  Shefner JM; Wolff AA; Meng L; Bian A; Lee J; Barragan D; Andrews JA; on behalf of the Benefit-ALS Study Group, Amyotroph Lateral Scler Frontotemporal Degener, 1/1/2016
• 215th ENMC International Workshop VCP-related multi-system proteinopathy (IBMPFD) 13-15 November 2015, Heemskerk, The Netherlands  
  Evangelista T, Weihl CC, Kimonis V, Lochm�ller H; VCP related diseases Consortium, Neuromuscul Disord, 1/1/2016
• Prospective exploratory muscle biopsy, imaging, and functional assessment in patients with late-onset Pompe disease treated with alglucosidase alfa: The EMBASSY Study  
  van der Ploeg A, Carlier PG, Carlier R-Y, Kissel RT, Schoser B, Wenninger S, Pestronk A, Barohn RJ, Dimachkie MM, Goker-Alpan O, Mozaffar T, Pena LDM, Simmons Z, Strau..., Mol Genet Metab, 1/1/2016
• Randomized Trial of Thymectomy in Myasthenia Gravis  
  Wolfe GI, Kamniski HJ, Aban IB, Minisman G, Kuo H-C, Marx A, Strobel P, Mazia C, Oger J, Cea JG, Heckmann JM, Evoli A, Nix W, Ciafaloni E, Antonini G, Witoonpanich R, ..., N Engl J Med, 1/1/2016
• ALSUntangled No 35: Hyperbaric Oxygen Therapy  
  Fixsen E, Bedlack R, Quinn C, et al., Amyotroph Lateral Scler Frontotemporal Degener, 1/1/2016
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Books/Book Chapters

• Unusual Diagnostic and Management Problems in Neuromuscular Disorders. 
  Chapter: Hereditary Myopathy with Early Respiratory Failure in a young Afghan woman. 
  Lai T, Goyal NA, Mozaffar TEditors: Connolly AM, Dyck PJ, Al-Lozi MT - course directors.American Academy of Neurology, Annual Meeting, Boston, MA2017
• Unusual Diagnostic and Management Problems in Neuromuscular Disorders 
  Chapter: Late onset ocular and proximal myopathy associated with myosin heavy chain 2 gene mutations. 
  Peyronnard, Goyal NA, Mozaffar TEditors: Connolly AM, Dyck PJ, Al-Lozi MT - course directors.American Academy of Neurology, Annual Meeting, Vancouver, BC (Canada)2016
• Unusual Diagnostic and Management Problems in Neuromuscular Disorders 
  Chapter: Hemibody weakness and hemibody atrophy in late onset myosin heavy chain 7 gene mutations. 
  Nayyar S, Mozaffar T, Goyal NAEditors: Connolly AM, Dyck PJ, Al-Lozi MT - course directors.American Academy of Neurology, Annual Meeting, Vancouver, BC (Canada)2016
• Unusual Diagnostic and Management Problems in Neuromuscular Disorders 
  Chapter: Adult onset LGMD with mutation in GMPBB gene. 
  Doan V, Goyal NA, Mozaffar TEditors: Connolly AM, Dyck PJ, Amato AA - course directors.American Academy of Neurology, Annual Meeting, Washington, DC2015
• Unusual Diagnostic and Management Problems in Neuromuscular Disorders 
  Chapter: Allgrove Syndrome. 
  Roy G, Goyal NA, Mozaffar TEditors: Connolly AM, Dyck PJ, Amato AA - course directors.American Academy of Neurology, Annual Meeting, Washington, DC2015
• Unusual Diagnostic and Management Problems in Neuromuscular Disorders 
  Chapter: Mitochondrial Depletion Syndrome. 
  Roy G, Goyal NA, Mozaffar TEditors: Connolly AM, Dyck PJ, Amato AA - course directors.American Academy of Neurology, Annual Meeting, Philadelphia, PA2014
• Unusual Diagnostic and Management Problems in Neuromuscular Disorders 
  Chapter: Myofibrillar Myopathy in a 5-year-old boy. 
  Cash TM, Goyal NA, Mozaffar TEditors: Connolly AM, Dyck PJ, Amato AA - course directors.American Academy of Neurology, Annual Meeting, Philadelphia, PA2014
• Unusual Diagnostic and Management Problems in Neuromuscular Disorders 
  Chapter: Neurolymphomatosis. 
  Santhappa S, Mozaffar TEditors: Meriggioli M, Dyck PJ, Amato AA - course directors.American Academy of Neurology, Annual Meeting, New Orleans2012
• Orthopaedic Knowledge Update 10. 
  Chapter: Neurological Disorders. 
  Page 225 - 238Mozaffar T and Gupta REditors: Fischgrund, J.American Academy of Orthopaedic Surgeons, Rosemont, IL2011
• Unusual Diagnostic and Management Problems in Neuromuscular Disorders 
  Chapter: HIBM-2/Nonaka Myopathy. 
  Mozaffar T, Wang AKEditors: Meriggioli M, Dyck PJ, Amato AA - course directors.American Academy of Neurology, Annual Meeting, Honolulu2011
• Disorders of Voluntary Muscle 
  Chapter: Endocrine and Toxic Myopathies (on line update). 
  Mozaffar TEditors: Karpati G, Hilton-Jones D, Bushby K and Griggs RC.Cambridge University PressEdition 8th,2010
• International Neurology: a clinical approach. 
  Chapter: Oculopharyngeal muscular dystrophy. 
  Page 231 - 232Chui LA, Mozaffar TEditors: Lisak RP, Truong DD, Carroll W, Bhidayasiri R.Wiley-Blackwell Publishing, Oxford, UK2009
• International Neurology: a clinical approach. 
  Chapter: Emery-Dreifuss muscular dystrophy. 
  Page 233 - 234Karayan R and Mozaffar TEditors: Lisak RP, Truong DD, Carroll W, Bhidayasiri R.Wiley-Blackwell Publishing, Oxford, UK2009
• Unusual Diagnostic and Management Problems in Neuromuscular Disorders 
  Chapter: X-linked myotubular myopathy in manifesting carriers. 
  Adzotor K, Mozaffar TEditors: Kissel JT, Jackson CE, Harati Y - course directors.American Academy of Neurology, Annual Meeting, Seattle2009
• Orthopedic Basic Science: Biology and Biomechanics of the Musculoskeletal System 
  Chapter: Peripheral Nerve Physiology, Anatomy, and Pathology. Chapter 23. 
  Page 427 - 443Gupta R, Mozaffar TEditors: Buckwalter JA, Einhorn TA, O�Keefe RJ.American Academy of Orthopedic Surgeons, Rosemont, ILEdition 3rd,2007
• Orthopedic Basic Science: Biology and Biomechanics of the Musculoskeletal System 
  Chapter: Form and Function of the Spinal Cord and Peripheral Nerves. Chapter 13. 
  Page 245 - 258Gupta R, Mozaffar TEditors: Buckwalter JA, Einhorn TA, O�Keefe RJ.American Academy of Orthopedic Surgeons, Rosemont, ILEdition 3rd,2007
• Unusual Diagnostic and Management Problems in Neuromuscular Disease 
  Chapter: Cervical Radiculopathy in Multiple Sclerosis. 
  Lueck KL, Mozaffar TEditors: Kissel JT, Jackson CE, Rosenfeld J - course directors.Academy of Neurology, Annual Meeting, San Diego, CA2006
• Unusual Diagnostic and Management Problems in Neuromuscular Disease 
  Chapter: Adrenomyeloneuropathy. 
  Lee KA, Mozaffar TEditors: Kissel JT, Jackson CE, Rosenfeld J - course directors.Academy of Neurology, Annual Meeting, San Diego, CA2006
• Unusual Diagnostic and Management Problems in Neuromuscular Disease 
  Chapter: Hereditary neuropathy with liability towards pressure palsies. 
  Mozaffar TEditors: Kissel JT, Jackson CE, Harati Y - course directors.Academy of Neurology, Annual Meeting, Miami Beach, FL2005
• Tropical Neurology 
  Chapter: Neuromuscular Complications of Malnutrition: Hansen�s Disease (Leprosy) 
  Mozaffar TEditors: Joseph Zunt, MD, Director.American Academy of Neurology Annual Meeting, Honolulu, HI2003
• Neuromuscular disease of infancy, childhood and adolescence: A clinician�s approach. 
  Chapter: Pediatric Leprosy. 
  Page 505 - 518Solbrig MV, Mozaffar T, Kim RCEditors: Jones HR, Darras BT, De Vivo DC.Butterworth-Heineman, Boston2003
• Structural and Molecular Basis of Skeletal Muscle Diseases 
  Chapter: Cancer-related muscle diseases. 
  Page 266 - 269Mozaffar T, Pestronk AEditors: Karpati G.ISN Neuropath Press, Basel2002
• The Washington Manual of Medical Therapeutics 
  Chapter: Neurologic emergencies. 
  Page 475 - 493Yamada K, Mozaffar T, Awadalla SEditors: Carey CF, Lee HH, Woeltje KF.Lippincott - Raven, PhiladelphiaEdition 29th,1998
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Abstracts/Posters

• Results from the MGTX Extension Study of thymectomy in myasthenia gravis.
  Wolfe G., Kaminski H., Aban I., Cutter G., MGTX Study Group, Muscle Nerve, 1/1/2018
• Results of an Investigator-Initiated Multi-Site LateOnset Pompe Disease Screening Study.
  Wencel M, Goyal N, Shaibani A, Dimachkie M, Johnson N, Trivedi J, Gutmann L, Wicklund M, Bandyopadhyay S, Genge A, Freimer M, Pestronk A, Florence J, Karam C, Ralph J,..., Ann Neurol, 1/1/2018
• Feasibility and validation of modified Oculobulbar facial respiratory score (mOBFRS) in sporadic inclusion body myositis.
  Wencel M, Araujo N, Medina E, Zhang LS, Nguyen D, Mozaffar T, Goyal N, Ann Neurol, 1/1/2018
• Feasibility and validation of modified Oculobulbar facial respiratory score (mOBFRS) in amyotrophic lateral sclerosis.
  Wencel M, Araujo N, Medina E, Zhang LS, Nguyen D, Mozaffar T, Goyal N, Ann Neurol, 1/1/2018
• Willingness to participate in natural history studies amongst patients with sporadic inclusion body myositis (sIBM): A comparison between the United States and Australia.
  Mathew V, Wencel M, Goyal N, Habib AA, Needham M, Mozaffar T, Muscle Nerve, 1/1/2018
• First in-human study of AT-GAA (ATB200/AT2221) in patients with Pompe Disease: Preliminary results from the ATB200-02 trial.
  Mozaffar T, Bratkovic D, Byrne BJ, Clemens P, Geberhiwot T, Goker-Alpan O, Kishnani P, Ming X, Roberts M, Schwenkreis P, Sivakumar K, van der Ploeg AT, Wright J, Johns..., Muscle Nerve, 1/1/2018
• Results of an Investigator-Initiated Multi-Site Late Onset Pompe Disease Screening Study.
  Wencel M, Goyal N, Shaibani A, Dimachkie M, Johnson N, Trivedi J, Gutmann L, Wicklund M, Bandyopadhyay S, Genge A, Freimer M, Pestronk A, Florence J, Karam C, Ralph J,..., Muscle Nerve, 1/1/2018
• Dalazatide, an Inhibitor of the Kv1.3 Channel on Chronically Activated Effector Memory T Cells, Has Immunotherapy Potential Against Sporadic Inclusion Body Myositis.
  Mozaffar T, Wencel M, Goyal NA, Phillips CW, Olsen C, Ann Neurol, 1/1/2017
• GNE-myopathy (HIBM): Upper and lower extremity muscle strength declines over time in a prospective.
  Behin A, Mozaffar T, Tarnopolsky M, et al., Neuromuscul Disord, 1/1/2017
• ActiMyo home monitoring in adult patients with limb girdle muscular dystrophy type 2B and facioscapulohumeral muscular dystrophy in study ATYR 1940-C-004.
  Gidaro T, Moraux A, Grelet M, et al., Neuromuscul Disord, 1/1/2017
• A phase 3 randomized, double blind, placebo-controlled study to evaluate the efficacy and safety of sialic acid extended-release tablets in patients with GNE myopathy ...
  Lochmuller H, Behin A, Caraco Y, et al., Neuromuscul Disord, 1/1/2017
• Kv1.3 expression on effector memory T cells in sporadic inclusion body myositis: potential for targeted immunotherapy with dalazatide.
  Mozaffar T, Wencel M, Goyal N, Philips C, Olsen C, Neuromuscul Disord, 1/1/2017
• Expanding importance of HMERF titinopathy: new mutations and clinical aspects.
  Palmio J, Leonard S, Sacconi S, et al., Neuromuscul Disord, 1/1/2017
• Results of a Phase 1b/2 Study of ATYR1940 in Adult Patients with Limb Girdle Muscular Dystrophy Type 2B (LGMD2B) and Facioscapulohumeral Muscular Dystrophy (FSHD) (ATY...
  Vissing J, Attarian S, Gidaro T, et al., Neurology, 1/1/2017
• Feasibility and validation of modified oculobulbar facial respiratory score (mOBFRS) in amyotrophic lateral sclerosis (ALS) and sporadic inclusion body myositis (sIBM).
  Wencel M, Araujo N, Mozaffar T, Goyal N, Neuromuscul Disord, 1/1/2017
• Feasibility and validation of modified oculobulbar facial respiratory score (mOBFRS) in amyotrophic lateral sclerosis (ALS) and sporadic inclusion body myositis (sIBM).
  Wencel M, Araujo N, Mozaffar T, Goyal N, Muscle Nerve, 1/1/2017
• Interim analysis of an investigator-initiated multi-site late-onset Pompe disease screening study.
  Wencel M, Goyal N, Mozaffar T, Neuromuscul Disord, 1/1/2017
• Challenges And Feasibility Of An Investigator-Initiated Multi-Site Pompe Disease Screening Study.
  Wencel M, Goyal N, Shaibani A, et al., Muscle Nerve, 1/1/2017
• Distal myopathy with associated hemiatrophy.
  Goyal N, Nayyar S, Mozaffar T, Neuromuscul Disord, 1/1/2016
• Response to IVIG in Patients with CIDP: The INSIGHTS Quality Improvement Project.
  Levine T, Katz J, Barohn R, et al., Ann Neurol, 1/1/2016
• Response To Intravenous Immunoglobulin In Patients With Chronic Inflammatory Demyelinating Polyneuropathy: The Insights Quality Improvement Project.
  Levine T, Katz J, Barohn R, et al., Muscle Nerve, 1/1/2016
• Insights: Analysis Of Ivig Responsiveness In Patients With Myasthenia Gravis.
  Levine T, Saperstein D, Vaughan L, et al., Muscle Nerve, 1/1/2016
• INSIGHTS: Analysis of IVIG Responsiveness in Patients with Myasthenia Gravis.
  Levine T, Vaughan L, Badger G, et al., Ann Neurol, 1/1/2016
• Insights: Analysis Of Intravenous Immunoglobulin Responsiveness In Patients With Myasthenia Gravis.
  Levine T, Vaughan L, Badger G, et al., Muscle Nerve, 1/1/2016
• Upper and lower extremity muscle strength decline over 1 year in a prospective, observational GNE-myopathy natural history study.
  Tarnopolsky M, Mozaffar T, Behin A, et al., Neuromuscul Disord, 1/1/2016
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Lectures

• ALS: Current Understanding of Biology and Treatment 
  Las Vegas, NV - 1/1/2018
• Therapeutic Advances in Myasthenia Gravis 
  Montreal Neurological Institute, Montreal, CA - 1/1/2018
• Update on Idiopathic Inflammatory Myopathies: Diagnosis and Treatment 
  Dallas, TX - 1/1/2018
• Utility of Genetic Testing in Neuromuscular Disorders 
  Vienna, Austria - 1/1/2018
• The Evolving Classification of Idiopathic Inflammatory Myopathies: Myopathologic and Serologic Advances 
  Los Angeles, CA - 1/1/2018
• Of Mice and Men: Lessons Learned from the Muscle Atrophy Inhibition Trials in Neurological Disorders 
  Sue and Bill Gross Stem Cell Center, University of California, Irvine - 1/1/2018
• Update on Myopathy Therapy 
  Oregon Health Sciences University, Portland, OR - 1/1/2018
• Respiratory Functions in Pompe Disease: Utility in Diagnosis and Monitoring of Treatment Response 
  New Orleans, LA - 1/1/2018
• Crossfire Session: Con: needle muscle biopsy is equally sufficient as open muscle biopsy 
  Phoenix, AZ - 1/1/2017
• VCP Associated Multisystem Proteinopathy 
  Singapore - 1/1/2017
• Antibody Testing in Inflammatory Myositides 
  Singapore - 1/1/2017
• Polymyositis: Fact or Fiction 
  Stony Brook University, Stony Brook, NY - 1/1/2017
• Unicorns, Dragons, Polymyositis, and Other Mythical Beasts: The Truth About Polymyositis 
  Boston, MA - 1/1/2017
• Introduction to Electrodiagnostic Studies 
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