TahseenMozaffarMD

PubMed

• 494 citations
  Efficacy of minocycline in patients with amyotrophic lateral sclerosis: a phase III randomised trial
  Paul H. Gordon, Dan H. Moore, Robert G. Miller, Julaine Florence, Joseph L. Verheijde
  The Lancet. Neurology. 2007-12-01
• 41 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
  The Lancet. Neurology. 2023-07-01
• 45 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
  Muscle & Nerve. 2022-03-01

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
• 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
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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
• 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
• 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
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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: 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: 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: 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: 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: 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: 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: Neurolymphomatosis. 
  Santhappa S, Mozaffar TEditors: Meriggioli M, Dyck PJ, Amato AA - course directors.American Academy of Neurology, Annual Meeting, New Orleans2012
• 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
• Orthopaedic Knowledge Update 10. 
  Chapter: Neurological Disorders. 
  Page 225 - 238Mozaffar T and Gupta REditors: Fischgrund, J.American Academy of Orthopaedic Surgeons, Rosemont, IL2011
• 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: Emery-Dreifuss muscular dystrophy. 
  Page 233 - 234Karayan R and Mozaffar TEditors: Lisak RP, Truong DD, Carroll W, Bhidayasiri R.Wiley-Blackwell Publishing, Oxford, UK2009
• 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
• 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: 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
• 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
• 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

• 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
• ALS: Current Understanding of Biology and Treatment 
  Las Vegas, NV - 1/1/2018
• Unicorns, Dragons, Polymyositis, and Other Mythical Beasts: The Truth About Polymyositis 
  Boston, MA - 1/1/2017
• Sporadic Inclusion Body Myositis: An Autoimmune vs. Myodegenerative Disease? 
  Northwell University, Great Neck, NY - 1/1/2017
• Clinical Research Studies in Myositis at UC Irvine 
  Orange, CA - 1/1/2017
• Neuromuscular Research Update 
  Oregon Health Sciences University, Portland, OR - 1/1/2017
• Introduction to Electrodiagnostic Studies 
  University of California, Riverside, Riverside, CA - 1/1/2017
• Polymyositis: Fact or Fiction 
  Stony Brook University, Stony Brook, NY - 1/1/2017
• Antibody Testing in Inflammatory Myositides 
  Singapore - 1/1/2017
• VCP Associated Multisystem Proteinopathy 
  Singapore - 1/1/2017
• Crossfire Session: Con: needle muscle biopsy is equally sufficient as open muscle biopsy 
  Phoenix, AZ - 1/1/2017
• Update on Treatment of Common Hereditary Neuromuscular Disorders 
  The Aga Khan University, Karachi, Pakistan - 1/1/2016
• Competency Based Neurology Residency Training: Opportunities and Way Forward 
  The Aga Khan University, Karachi, Pakistan - 1/1/2016
• Myasthenia Gravis: What is New in Research and What is on the Horizon? 
  Houston, TX - 1/1/2016
• Conventional Treatment of Immune Mediated Necrotizing Myopathies 
  Zandvoort, Netherlands - 1/1/2016
• Pompe Disease: Day 0 through Day 30000 
  Arlington, VA - 1/1/2016
• Monitoring for Disease Manifestation in Late-Onset Pompe Disease: Challenges from the Newborn Screening Program 
  St. Louis, MO - 1/1/2016
• Pompe Disease: When Limb-Girdle Muscle Weakness Means Something Out of the Ordinary 
  Hennepin County Medical Center, Minneapolis, MN - 1/1/2015
• Pompe Disease Update 
  Hershey, PA - 1/1/2015
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