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.
May 16th 2025
Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending May 16, 2025.
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
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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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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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Identifying and Treating CIDP in the Modern Era
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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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New Treatments, Global Collaboration Highlight 2025 Peripheral Nerve Society Annual Meeting
May 13th 2025Committee chairs Vincent Timmerman, PhD, MSc, and Kathrin Doppler, MD, provide a preview of the 2025 PNS Annual Meeting, giving clinicians an inside look at what to expect from the upcoming conference.
Muscle MRI May Serve as Sensitive Biomarker for ALS Disease Progression
May 8th 2025A study involving 20 patients with ALS found that longitudinal muscle MRI detected progressive atrophy and correlated with muscle strength loss, supporting its potential as a biomarker for ALS progression and clinical trials.
Gene-Targeted Therapies and Biomarkers Driving ALS Research Forward: Brian Lin, PhD
May 4th 2025The research portfolio director at the Muscular Dystrophy Association talked about recent advances in ALS research highlight the promise of gene-targeted treatments and the increasing utility of biomarkers for diagnosis. [WATCH TIME: 3 minutes]
Honoring 2025 SEQUINS Hall of Famers: Perspectives From Cheryl Bushnell, MD, MHS
May 1st 2025Cheryl Bushnell, MD, MHS, a professor of neurology at Wake Forest University, discussed her SEQUINS Hall of Fame recognition, her work on sex differences in stroke risk, and her ongoing efforts to improve stroke care equity.
FDA Approves FcRn Blocker Nipocalimab for Broad Forms of Generalized Myasthenia Gravis
April 30th 2025Administered intravenously in a single-dose vial, nipocalimab becomes the second FDA-approved therapy for common forms of generalized myasthenia gravis, and the first for younger patients living with the disease.
Durability and Next Steps in Advancing ARCUS Gene Editing Platform in DMD: Cassandra Gorsuch, PhD
April 26th 2025The chief scientific officer at Precision Biosciences provided clinical insights on the durable potential of ARCUS gene editing and the company’s next steps toward in-human trials for Duchenne muscular dystrophy. [WATCH TIME: 4 minutes]
Unveiling ARCUS Gene Editing Platform and Its Potential in DMD: Cassandra Gorsuch, PhD
April 23rd 2025The chief scientific officer at Precision Biosciences provided clinical commentary on ARCUS gene editing platform and its potential application in Duchenne muscular dystrophy through a durable, AAV-delivered therapeutic strategy. [WATCH TIME: 4 minutes]
Final CHAMPION MG Trial Data Show Sustained Benefit, Safety of Ravulizumab in Myasthenia Gravis
April 22nd 2025Results from the open-label extension of a phase 3 trial showed that treatment with ravulizumab sustained clinical benefits for up to 4 years in patients with generalized myasthenia gravis.
Breaking Down Oculopharyngeal Muscular Dystrophy: From Clinical Gaps to Genetic Gains
April 22nd 2025Matthew Wicklund, MD, a professor of neurology at the University of Texas Health Science Center San Antonio, offered a clinical and translational overview of OPMD, highlighting current care challenges and the early promise of dual-action gene therapy strategies.
From Fibrosis to Function: Exploring New Pathways in Muscular Dystrophy Research
April 19th 2025Neuromuscular expert Matthew Alexander, PhD, explores the evolving landscape of targeted therapies in muscular dystrophies, from fibrosis and glycosylation to combination strategies with gene therapy.