Continuum: Lifelong Learning in Neurology—Muscle and Neuromuscular Junction Disorders, Volume 22, Issue 6, December 2016
Muscle and Neuromuscular Junction Disorders December 2016;22(6)
Continuum: Lifelong Learning in Neurology® is designed to help practicing neurologists stay abreast of advances in the field while simultaneously developing lifelong self-directed learning skills.
Upon completion of this Continuum: Lifelong Learning in Neurology Muscle and Neuromuscular Junction Disorders issue, participants will be able to:
• Utilize electrodiagnostic tests in the assessment of patients with suspected muscle disease, describe the uses of imaging to distinguish different forms of muscle disease and exclude mimics, and recognize the various indications for muscle biopsy
• Demonstrate a focused approach to the clinical assessment and investigation of the patient with hyperCKemia by considering common etiologies, including those that are potentially life limiting or may change management
• Recognize and describe the clinical features and management of endocrine myopathies and of myopathies due to medications or other toxins
• Develop a diagnostic and treatment algorithm for the metabolic myopathies
• Diagnose and treat different subtypes of autoimmune myopathy
• Recognize the clinical features and discuss the pathogenesis of inclusion body myositis
• Diagnose and manage the dystrophic and nondystrophic myotonias
• Diagnose and manage patients with facioscapulohumeral muscular dystrophy
• Recognize the key features and classification of congenital myopathies, understand recent advances in molecular genetics and the use of muscle imaging for the diagnosis of congenital myopathies, and develop a multidisciplinary approach to the treatment of congenital myopathies
• Recognize the phenotypic presentation and approach to the evaluation of the various limb-girdle muscular dystrophies
• Describe the neurophysiology of neuromuscular transmission and the pathophysiology of its disorders, and diagnose and manage acquired disorders of neuromuscular transmission, including autoimmune myasthenia gravis and the Lambert-Eaton myasthenic syndrome
• Apply ethical principles in providing genetic counseling for asymptomatic individuals at risk for progressive neuromuscular disorders
• Recognize the strengths and limitations of patient registries while reviewing strategies to discuss patient registries with patients
This Continuum: Lifelong Learning in Neurology Muscle and Neuromuscular Junction Disorders issue covers the following core competencies:
• Patient Care and Procedural Skills
• Medical Knowledge
• Practice-Based Learning and Improvement
• Interpersonal and Communication Skills
• Systems-Based Practice
Hannah R. Briemberg, MD, FRCPC, Guest Editor
Clinical Associate Professor of Neurology, University of British Columbia, Vancouver, British Columbia, Canada
aDr Briemberg serves on the editorial board of Continuum and has received personal compensation for serving as a consultant for Novartis AG and also has received publishing royalties from BMJ Publishing Group Ltd and UpToDate, Inc. Dr Briemberg has received research support related to the Canadian Neuromuscular Disease National Registry from the University of Calgary and has received research/grant support as medical director of the amyotrophic lateral sclerosis clinic from the Amyotrophic Lateral Sclerosis Society of British Columbia.
bDr Briemberg discusses the unlabeled/investigational use of immunosuppressive medications for the treatment of autoimmune myositis.
Anthony A. Amato, MD, FAAN
Vice Chairman, Department of Neurology, Brigham and Women’s Hospital; Chief, Neuromuscular Division, Brigham and Women’s Hospital; Professor of Neurology, Harvard Medical School, Boston, Massachusetts
aDr Amato has received personal compensation for serving on the medical advisory board of and as a consultant for Akashi Therapeutics, CSL Behring, and Novartis AG and for serving as associate editor of Muscle & Nerve and Neurology. Dr Amato has received research/grant support as site principal investigator for Alexion Pharmaceuticals, Inc; the National Institutes of Health; and Novartis AG; has received publishing royalties from UpToDate, Inc; and has provided expert witness testimony related to a wrongful diagnosis legal case.
bDr Amato reports no disclosure.
Steven A. Greenberg, MD
Associate Professor of Neurology, Brigham and Women’s Hospital and Children’s Hospital; Associate Professor of Neurology, Harvard Medical School, Boston, Massachusetts
aDr Greenberg has served as a consultant for Acceleron Pharma and Novartis AG and receives research/grant support from the Inclusion Body Myositis Foundation, Inc and Pfizer Inc. Dr Greenberg receives licensing fees from MedImmune and publishing royalties from UpToDate, Inc.
bDr Greenberg reports no disclosure.
Stanley Jones P. Iyadurai, MSc, PhD, MD
Assistant Professor of Neurology and Neuromuscular Medicine, Ohio State University, Columbus, Ohio
aDr Iyadurai has received personal compensation for serving on the advisory boards for Allergan; Alnylam Pharmaceuticals, Inc; CSL Behring; and Pfizer, Inc.
bDr Iyadurai discusses the unlabeled/investigational use of corticosteroids to treat Duchenne muscular dystrophy.
Lyell K. Jones Jr, MD, FAAN
Associate Professor of Neurology; Neurology Residency Program Director, Department of Neurology, Mayo Clinic, Rochester, Minnesota
aDr Jones has received publishing royalties from the Mayo Clinic Neurology Board Review.
bDr Jones reports no disclosure.
Jeffrey T. Joseph, MD, PhD
Professor, Neuropathology Group Director, Department of Pathology and Laboratory Medicine, Calgary Laboratory Services; Donald Burns and Louise Berlin Professorship in Dementia Research, Hotchkiss Brain Institute, University of Calgary, Calgary, Alberta, Canada
aDr Joseph has received research/grant support from the Endowed Chair of the University of Calgary to create the brain tissue bank and receives publishing royalties from UpToDate, Inc and Wolters Kluwer. Dr Joseph also gave expert testimony for Alberta Justice, for which he did not receive compensation.
bDr Joseph reports no disclosure.
Charles D. Kassardjian, MD
Neurologist, Saint Michael’s Hospital; Lecturer, University of Toronto, Toronto, Ontario, Canada
aDr Kassardjian has received honoraria for grand rounds lectures for Sanofi Genzyme.
bDr Kassardjian reports no disclosure.
Hans D. Katzberg, MD
Associate Professor of Medicine, University of Toronto, Toronto, Ontario, Canada
aDr Katzberg has received personal compensation for serving as a consultant for Flex Pharma and Octapharma AG, for serving as a speaker and a consultant for Sanofi Genzyme, and for serving on the advisory board of and as a consultant and a speaker for CSL Behring and Grifols. Dr Katzberg has received research grants from CSL Behring, Grifols, and Muscular Dystrophy Canada.
bDr Katzberg reports no disclosure.
John T. Kissel, MD, FAAN
Chair, Department of Neurology, Pediatrics, and Neuroscience, Ohio State University Wexner Medical Center, Columbus, Ohio
aDr Kissel has received personal compensation for serving on a consulting board of AveXis, Inc; as journal editor of Muscle & Nerve; and as a consultant for Novartis AG. Dr Kissel has received research/grant funding as principal investigator of a study from the National Institutes of Health and has received funding for clinical trials from AveXis, Inc; AxelaCare Health solutions, LLC; BioMarin; CSL Behring; Cytokinetics, Inc; Ionis Pharmaceuticals; and Quintiles, Inc; and receives publishing royalties from Oxford University Press.
bDr Kissel discusses the unlabeled/investigational use of corticosteroids to treat Duchenne muscular dystrophy.
Jean K. Mah, MD, MSc, FRCPC
Associate Professor, Departments of Pediatrics and Clinical Neuroscience, Alberta Children’s Hospital Research Institute, Cumming School of Medicine, University of Calgary, Calgary, Alberta, Canada
aDr Mah has received personal compensation as a consultant for aTyr Pharma and PTC Therapeutics and has received research/grant support as study and site investigator for Alberta Children’s Hospital, Bristol-Myers Squibb, Children’s Hospital of Eastern Ontario, Cooperative International Neuromuscular Research Group, Eli Lilly and Company, FSHD Global Research Foundation, FSH Society, the Hospital for Sick Children, Muscular Dystrophy Canada, Novartis AG, Pfizer Inc, PTC Therapeutics, and Sanofi Genzyme.
bDr Mah reports no disclosure.
Andrew L. Mammen, MD, PhD
Investigator, National Institute of Arthritis and Musculoskeletal and Skin Diseases, National Institutes of Health, Bethesda, Maryland; Adjunct Professor of Neurology and Medicine, Johns Hopkins University School of Medicine, Baltimore, Maryland
aDr Mammen receives intramural research funding from the National Institutes of Health.
bDr Mammen discusses the unlabeled/investigational use of azathioprine, cyclophosphamide, cyclosporine, IV immunoglobulin, methotrexate, methylprednisolone, mycophenolate mofetil, prednisone, rituximab, and tacrolimus for the treatment of autoimmune myopathies.
John P. Ney, MD, MPH
Assistant Professor of Neurology, Boston University School of Medicine, Boston, Massachusetts; Adjunct Faculty, Health Policy and Management, School of Public Health, Indiana University, Indianapolis, Indiana
aDr Ney has received personal compensation as an editorial board member of Neurology and as a consultant for Alliance Life Sciences Consulting Group Inc, AxelaCare, and SpecialtyCare, and has also given expert medical testimony in a court deposition. Dr Ney is a current employee of the United States government, and this manuscript was not a term of his employment nor did he receive any compensation for the manuscript.
bDr Ney reports no disclosure.
Michael W. Nicolle, MD
Director, Myasthenia Gravis Clinic; Chief, Division of Neurology, Department of Clinical Neurological Sciences; Director, EMG Laboratory and Neuromuscular Group, Schulich School of Medicine and Dentistry, Western University, London, Ontario, Canada
aDr Nicolle has given expert medical testimony in court cases for the Canadian Medical Protective Association.
bDr Nicolle discusses the unlabeled/investigational use of azathioprine, mycophenolate mofetil, and rituximab for the treatment of myasthenia gravis and Lambert-Eaton myasthenic syndrome and the use of 3,4-diaminopyridine for the treatment of Lambert-Eaton myasthenic syndrome.
Laura K. Rosow, MD
Associate Director, ALS Center; Assistant Professor of Neurology, University of California San Francisco, San Francisco, California
a,bDr Rosow reports no disclosures.
Valeria A. Sansone, MD
Clinical Director, Centro Clinico Nemo; Faculty, Neurology Department, University of Milan, Milan, Italy
aDr Sansone has received research support from Telethon (Unione Italiana Lotta alla Distrofia Musculare [UILDM] grant GUP15004).
bDr Sansone discusses the unlabeled/investigational use of clomipramine, flecainide, mipramine, mexiletine, and tocainide for the treatment of myotonia; acetazolamide and dichlorphenamide for the treatment of periodic paralysis; creatinine and tricyclic antidepressants for the treatment of muscle pain; and modafinil for the treatment of fatigue in myotonic dystrophy.
Anant M. Shenoy, MD
Assistant Professor of Neurology, Tufts University School of Medicine, Boston, Massachusetts; Attending in Neurology, Baystate Health, Springfield, Massachusetts
a,bDr Shenoy reports no disclosures.
Zachary Simmons, MD, FAAN
Professor of Neurology and Humanities, Pennsylvania State Milton S. Hershey Medical Center, Pennsylvania State University, Hershey, Pennsylvania
aDr Simmons serves on the editorial board of Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration and has received personal compensation as senior associate editor of Muscle & Nerve and for serving as a consultant for the ALS Association Greater Philadelphia Chapter; Cytokinetics, Inc; and Neuralstem, Inc. Dr Simmons has received research grant support from the ALS Association; Biogen; Cytokinetics, Inc;and Sanofi Genzyme.
bDr Simmons reports no disclosure.
Jeffrey M. Statland, MD
Assistant Professor of Neurology, University of Kansas Medical Center, Kansas City, Kansas
aDr Statland received personal compensation for serving on the advisory boards of Bristol-Myers Squibb and Sarepta Therapeutics and for serving as a consultant for Acceleron Pharma, Clinical Leader, and Novartis AG. Dr Statland has received research/grant support from the FSH Society and the National Center for Advancing Translational Sciences through the Clinical and Translational Science Award (KL2TR000119) and the Multi-institution Clinical and Translational Science Award.
bDr Statland reports no disclosure.
Mark A. Tarnopolsky, MD, PhD
Professor of Pediatrics and Medicine, Division of Neuromuscular and Neurometabolic Disorders, McMaster University, Hamilton, Ontario, Canada
aDr Tarnopolsky has received personal compensation as a speaker and consultant for Sanofi Genzyme and has received research funding from the Canadian Institutes of Health Research, Exerkine Corporation, and MitoCanada. Dr Tarnopolsky is also a shareholder and stockholder for Exerkine Corporation.
bDr Tarnopolsky discusses the unlabeled/investigational use of triheptanoin, which is under license and investigation by Ultragenyx Pharmaceutical, Inc, to treat fatty acid oxidation defects.
Rabi Tawil, MD, FAAN
Professor of Neurology, Pathology and Laboratory Medicine, University of Rochester Medical Center, Rochester, New York
aDr Tawil has received personal compensation as a consultant for Acceleron Pharma; aTyr Pharma; Novartis AG; and Third Rock Ventures, LLC and has received research/grant support as co-investigator of studies for the National Institutes of Health (U01 NS061795-04, 1U54AR065139, 1P01 NS069539) and as investigator of studies for the FSH Society and Northwest Friends of FSH Research. Dr Tawil receives publishing royalties from Wiley-Blackwell.
bDr Tawil reports no disclosure.
Shannon L. Venance, MD, PhD, FRCPC
Associate Professor, Western University, Department of Clinical Neurological Sciences, Schulich School of Medicine and Dentistry, London, Ontario, Canada
aDr Venance has received personal compensation for serving on the speaker’s bureau of and giving lectures related to muscle disease CME for Sanofi Genzyme Canada and has received publishing royalties from John Wiley & Sons, Inc.
bDr Venance reports no disclosure.
Ronnie Bergen, MD
Assistant Professor of Clinical Neurology, University of Arizona; Staff Neurologist, Southern Arizona Veterans Affairs Health Care System, Tucson, Arizona
a,bDr Bergen reports no disclosure.
Douglas J. Gelb, MD, PhD, FAAN
Professor of Neurology, University of Michigan, Ann Arbor, Michigan
aDr Gelb receives royalties from Oxford University Press and UpToDate, Inc.
bDr Gelb reports no disclosure.
Methods of Participation and Instructions for Use
Continuum: Lifelong Learning in Neurology® is designed to help practicing neurologists stay abreast of advances in the field while simultaneously developing lifelong self-directed learning skills. In Continuum, the process of absorbing, integrating, and applying the material presented is as important as, if not more important than, the material itself.
The goals of Continuum include disseminating up-to-date information to the practicing neurologist in a lively, interactive format; fostering self-assessment and lifelong study skills; encouraging critical thinking; and, in the final analysis, strengthening and improving patient care.
Each Continuum issue is prepared by distinguished faculty who are acknowledged leaders in their respective fields. Six issues are published annually and are composed of review articles, case-based discussions on ethical and practice issues related to the issue topic, coding information, and comprehensive CME and self-assessment offerings, including a selfassessment pretest, multiple-choice questions with preferred responses, and a patient management problem. For detailed instructions regarding Continuum CME and self-assessment activities, visit aan.com/continuum/cme.
The review articles emphasize clinical issues emerging in the field in recent years. Case reports and vignettes are used liberally, as are tables and illustrations. Video material relating to the issue topic accompanies issues when applicable.
The text can be reviewed and digested most effectively by establishing a regular schedule of study in the office or at home, either alone or in an interactive group. If subscribers use such regular and perhaps new study habits, Continuum’s goal of establishing lifelong learning patterns can be met.