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.
April 19th 2025
Backed by promising early data and mechanistic insights, SNUG01 enters clinical trials as the first TRIM72-based gene therapy candidate for ALS.
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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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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NeurologyLive® Brain Games: September 25, 2022
September 25th 2022Test your neurology knowledge with NeurologyLive®'s weekly quiz series, featuring questions on a variety of clinical and historical neurology topics. This week's topic is the history of the American Association of Neuromuscular & Electrodiagnostic Medicine.
Unique Composition of CNM-Au8 to Treat MS and ALS: Michael Hotchkin
September 24th 2022The chief development officer at Clene Nanomedicine provided insight on why CNM-Au8, an investigational agent in development, has shown positive results across both multiple sclerosis and ALS. [WATCH TIME: 2 minutes]
Eplontersin Treatment Results in Reduction in Serum Transthyretin in Amyloidosis
September 23rd 2022Eplontersen treatment resulted in a significant reduction in transthyretin, neuropathy impairment, and improvement in quality of life in the phase 3 NEURO-TTRansform study of patients with hereditary transthyretin amyloidosis.
Rituximab Reduces Probability of Myasthenia Gravis Disease Manifestation in New-Onset Disease
September 20th 2022A single, 500-mg intravenous dose of rituximab in individuals with new-onset generalized myasthenia gravis reduced the risk of disease manifestation and the need for rescue medications.
Human Endogenous Retroviruses and Their Role in ALS: Avindra Nath, MD
September 19th 2022The clinical director of the National Institute of Neurological Disorders and Stroke provided context on two recent papers that highlight a reactivation of HERV-K virus in the development of amyotrophic lateral sclerosis. [WATCH TIME: 5 minutes]
Celebrating the Expansion and Stressing the Importance of Newborn Screening
September 17th 2022The explosion of new science and legislative decisions have expanded the capabilities and reach of newborn screening, providing thousands of families every year an opportunity to seek early, and critical, care for their infants.
Future Implications, Outlook of New CDC Developmental Milestone Guidelines: Paul Lipkin, MD
September 13th 2022The director of medical outpatient services at the Kennedy Krieger Institute discussed the long-term effects of the updated developmental milestone guidelines and the need to judge children based on the 75th percentile. [WATCH TIME: 7 minutes]
FDA Accepts Application for Trofinetide in Rett Syndrome
September 12th 2022The treatment, previously known as NNZ-2566, is supported by data from the LAVENDER study. With no plans to hold an advisory meeting, the agency set a PDUFA date of March 12, 2023, for the Acadia Pharmaceuticals product.