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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Transition of Care in Young Adults With Spinal Muscular Atrophy
Medical professionals explore the challenges and considerations associated with patients with spinal muscular atrophy (SMA) transitioning to independent living, emphasizing the importance of maintaining continuity of care and addressing potential adherence issues to ensure optimal disease management.
Real-World Data for Risdiplam in Adult Patients with Spinal Muscular Atrophy
Panelists examine the available real-world data (RWD) on the use of risdiplam in adult patients with spinal muscular atrophy (SMA) from various studies conducted worldwide, discussing its potential efficacy and safety as a treatment option for adult SMA patients.
Updates in Therapeutic Development: Clinical Trial Readouts to Watch in the Second Half of 2024
July 17th 2024Take a look at some of the most-anticipated clinical trials with expected data readouts in the second half of 2024 that researchers and clinicians in neurology should keep an eye out on.
NeuroVoices: Marie Eugene, DO, on Career Development for Clerkship Directors
Published: July 17th 2024 | Updated: July 17th 2024The neurologist and clerkship director at the University of Connecticut provided perspective on the developing role of clerkship directors and the value they provide to neurology departments.
Real-World Data and Stability with Nusinersen in Adult Patients with Spinal Muscular Atrophy
Key opinion leaders address the scarcity of randomized controlled trial data for SMA treatments in adult populations, highlighting the availability of real-world data from numerous studies and provide guidance on how to incorporate this information when making treatment decisions for adult patients with spinal muscular atrophy.
Counseling Patients About Treatment Outcomes in Spinal Muscular Atrophy
Panelists delve into strategies for engaging in conversations with patients about defining and identifying successful therapy outcomes, providing guidance on how to effectively communicate and establish realistic expectations for treatment success in the context of spinal muscular atrophy (SMA).
Determining Treatment Success in Spinal Muscular Atrophy
Panelists share their approaches to counseling adult patients with spinal muscular atrophy (SMA) about treatment expectations.
Switching Treatments in Spinal Muscular Atrophy
Key opinion leaders share their strategies for discussing potential changes in therapy and transitioning between treatments with patients, emphasizing the importance of reassuring patients that such changes are intended to provide long-term benefits for managing their spinal muscular atrophy.