Neuromuscular
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.
Latest News
April 3rd 2025
The European Medicines Agency has requested a pause on 3 clinical trials of FDA-approved gene therapy Elevidys following the death of a patient who suffered acute liver failure after treatment.
Burst Expert Illustrations & Commentary™: Visualizing the Role of FcRN in Neurological Disorders
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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 and Commentaries™: Visualizing the Implications of FcRN-Targeted Therapies on Generalized Myasthenia Gravis
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Clinical Consultations™: Navigating the Evolving Treatment Landscape in Generalized Myasthenia Gravis
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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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SimulatED™: Diagnosing and Treating Alzheimer’s Disease in the Modern Era
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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 Disease Summit
September 27, 2025
Register Now!
SimulatEd™ From Discomfort to Relief: Acute Pain Management Essentials
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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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Medical Crossfire®: Integrating Real-World Data to Improve Outcomes for Patients With Multiple Sclerosis
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Medical Crossfire® - Optimizing Management for Patients With Generalized Myasthenia Gravis: Focus on Complement Inhibitors
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Burst CME™: Setting the Stage – Individualizing Migraine Care for Diverse Populations Across Care Settings
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Burst CME™: The Patient Journey – Unmet Needs From Diagnosis Through Management of Migraine
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Evolving Perspectives in Alzheimer Disease : Reaching an Earlier Diagnosis, Understanding Neuroinflammation, and Exploring Therapeutic Advances
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Burst CME™: Optimizing the Use of CGRP Targeted Agents for the Treatment of Migraine
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Burst CME™: Optimizing Migraine Management – Addressing Unmet Needs, Individualizing Care for Diverse Populations, and Utilizing CGRP Targeted Agents
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Community Practice Connections™: Optimizing the Management of Tardive Dyskinesia—Addressing the Complexity of Care With Targeted Treatment
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Cases and Conversations™: Applying Best Practices to Prevent Shingles in Your Practice
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Patient, Provider, and Caregiver Connection™: Pediatric Myasthenia Gravis—Current Treatment and Emerging Concepts
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Mind Moments™: Optimizing Management for Patients with Generalized Myasthenia Gravis
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Identifying and Treating CIDP in the Modern Era
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Cases and Conversations™: Navigating the Complexities of Managing Myasthenia Gravis in Pediatric and Pregnant Patient Populations
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Cases and Conversations™: Little Voices, Big Challenges – Comprehensive Care for Pediatric Spinal Muscular Atrophy
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A Breath of Strength: Managing Cancer Associated LEMS and Lung Cancer as One
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Striking the Right Nerve: Managing Cancer Associated LEMS in Lung Cancer Patients
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More News
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.
The State of Charcot-Marie-Tooth Disease: An In-Depth Interview With K. Florian P. Thomas, MD
May 24th 2019The Director of the Multiple Sclerosis and Hereditary Neuropathy Centers at Seton Hall-Hackensack Meridian School of Medicine shared insight into what clinicians should know about Charcot-Marie-Tooth disease.
Amit Rakhit, MD, MBA: Measuring Improvement in Fragile X Syndrome, Other Rare Conditions
May 24th 2019The chief medical officer and head of Research & Development at Ovid Therapeutics spoke about what prompted the development of this scale to measure improvement for patients with Fragile X syndrome, and possibly other conditions.
Muscular Dystrophy Association Panel Calls for Expanded Newborn Screening
May 23rd 2019An MDA panel has stated that accessibility of advanced screening, the introduction of effective treatment, and the support of professional organizations could, and should, prompt the expansion of newborn screening.
Amifampridine Gets Go-Ahead as LEMS Treatment in Pediatric Patients
May 13th 2019The amifampridine tablets are the first approved therapy specifically indicated for the treatment of pediatric patients with LEMS, a rare autoimmune disorder with neuromuscular symptoms caused by affected nerve connections and muscle weakness.
Eculizumab Significantly Reduces Relapse Risk in Neuromyelitis Optica Spectrum Disorder
May 7th 2019The treatment was efficacious in patients with AQP4-IgG-positive NMOSD, which accounts for nearly three-quarters of the population with the disorder, for which there is currently no approved treatment.
