The NeurologyLive® neuromuscular disease clinical focus page offers updates and information related to the clinical care and management of individuals with neuromuscular diseases such as amyotrophic lateral sclerosis, Duchenne muscular dystrophy, cerebral palsy, and spinal muscular atrophy, as well as rare diseases. This page consists of news, podcasts, and videos about the latest FDA actions, clinical guideline updates, interviews with physicians, study and clinical trial findings, and more.
December 5th 2024
RGX-202 demonstrated improved functional outcomes and robust microdystrophin expression, with levels exceeding those of other approved or investigational gene therapies.
5th Annual International Congress on the Future of Neurology®
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Clinical Consultations™: Managing Depressive Episodes in Patients with Bipolar Disorder Type II
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Advances In™ Generalized Myasthenia Gravis: Improving Patient Outcomes Through Early Diagnosis and Management
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Medical Crossfire®: Understanding the Advances in Bipolar Disease Treatment—A Comprehensive Look at Treatment Selection Strategies
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'REEL’ Time Patient Counseling: The Diagnostic and Treatment Journey for Patients With Bipolar Disorder Type II – From Primary to Specialty Care
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Community Practice Connections™: Transforming Multiple Sclerosis Care – Clinical Updates on the Effects of BTK Inhibitors
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Burst CME: Optimizing Therapy in Parkinson’s Disease
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Burst Expert Illustrations & Commentary™ : Visualizing the Role of the Complement Proteins in Neurologic Disorders
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Addressing Healthcare Inequities: Bridging the Gap in Multiple Sclerosis – A Focus on Clinical and Healthcare Disparities in Black Patients
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Burst Expert Illustrations & Commentary™: Visualizing the Implications of Anti-Complement Therapies on Generalized Myasthenia Gravis
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Burst Expert Illustrations & Commentary™: Visualizing the Role of the Complement Pathway in Neurological Disorders
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Little Voices, Big Challenges: Comprehensive Care for Pediatric Spinal Muscular Atrophy
January 9, 2025
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Individualizing Treatment for Patients with Generalized Myasthenia Gravis
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Patient, Provider & Caregiver Connection™: Reducing the Burden of Parkinson Disease Psychosis with Personalized Management Plans
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Clinical ShowCase™ in ALS: Addressing Diagnostic Delays, Evolving Therapies, and Multidisciplinary Care
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BURST Expert Illustrations and Commentaries™: Visualizing FcRn as a Therapeutic Target in Neurological Disease
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Burst Expert Illustrations & Commentary™: Visualizing the Role of FcRN in Neurological Disorders
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BURST Expert Illustrations and Commentaries™: Visualizing the Implications of FcRN-Targeted Therapies on Generalized Myasthenia Gravis
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SimulatED™: Diagnosing and Treating Alzheimer’s Disease in the Modern Era
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Burst Expert Illustrations & Commentary™: Visualizing the Role of Subcutaneous Infusion as an Alternate Administration Route for Medical Interventions
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Clinical Consultations™: Navigating the Evolving Treatment Landscape in Generalized Myasthenia Gravis
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SimulatED™: Understanding the Role of Genetic Testing in Patient Selection for Anti-Amyloid Therapy
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Expert Illustrations & Commentaries™: New Targets for Treatment in Cognitive Impairment in Schizophrenia – The Role of NMDA Receptors and Co-agonists
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BURST CME™ Part I: Understanding the Impact of Huntington’s Disease
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Burst CME™ Part II: The Evolving Treatment Landscape for Huntington Disease
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Carolina Neuromuscular Summit
September 27, 2025
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Evolving Perspectives in Alzheimer's Disease: Reaching an Earlier Diagnosis, Understanding Neuroinflammation, and Exploring Therapeutic Advances
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Clinical ShowCase: Developing a Personalized Treatment Plan for a Patient with Huntington’s Disease Associated Chorea
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SimulatEd™ From Discomfort to Relief: Acute Pain Management Essentials
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Medical Crossfire® - Optimizing Management for Patients With Generalized Myasthenia Gravis: Focus on Complement Inhibitors
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FDA Cites Active Ingredient Issue With Riluzole For Treatment of Amyotrophic Lateral Sclerosis
July 22nd 2019The complete response letter cited issues with an active pharmaceutical ingredient used in the Biohaven 2017 bioequivalence study that was manufactured between 2014 and 2016 in an Apotex facility.
Future of Neurology Congress Will Offer Expert Insights on Hot Topics
July 15th 2019The International Congress on the Future of Neurology will take place this Fall in New York City, converging experts in neurology to discuss the latest data and best practices to better inform clinical decision-making.
Identifying Myasthenia Gravis Earlier in Patients Who Fail to Seek Treatment
July 2nd 2019The clinical assistant professor of neurology and neurosurgery at the University of Texas discussed the need to identify men who may be at risk for myasthenia gravis despite a lack of willingness to see their physician.
Investigational DMD Gene Therapy Reports Positive Preliminary Results in Phase 1b
July 1st 2019PF-06939926 is a recombinant adenoassociated virus serotype 9 (AAV9) capsid carrying mini-dystrophin, a shortened version of the human dystrophin gene, under the control of a muscle-specific promoter.
Addressing Serious Neurologic Conditions in Men
June 28th 2019The clinical assistant professor of neurology and neurosurgery at the University of Texas spoke about the staggering number of men who forgo seeing physicians for serious conditions and how the clinical community can help address the challenge.
Advocacy Groups Look Toward Increasing Support for Providers in Rare Neurological Disorders
May 25th 2019The National Organization for Rare Disorders is home base for more than 300 member organizations that serve not just patients, but providers who care for those with rare disorders, such as SMA and Duchenne muscular dystrophy.