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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NeuroVoices: Benzi Kluger, MD, MS, on the Expansion and Growth of Neuropalliative Care
November 23rd 2022The president of the International Neuropalliative Care Society provided perspective on the growth of the field of neuropalliative care, the organization’s role, and how clinicians are overcoming current barriers in place.
Growth of Neuropallaitive Care and the Bright Future: Benzi Kluger, MD, MS
November 22nd 2022The president of the International Neuropalliative Care Society discussed the area in which neuropalliative has expanded and improved, as well as the potential in the coming years. [WATCH TIME: 2 minutes]
Post-Conference Perspectives and Updates in Neuropalliative Care: Benzi Kluger, MD, MS
November 21st 2022The president of the International Neuropalliative Care Society provided an overview of the organization’s recently concluded conference and the significance of the growth of neuropalliative care. [WATCH TIME: 6 minutes]
Rituximab Significantly Lessens Myasthenia Gravis Manifestations, Rescue Treatments Needed
November 18th 2022Compared with placebo, a greater proportion of patients on rituximab met the primary end point of minimal disease manifestations at 16 weeks, and demonstrated favorable results on several secondary end points.
NeuroVoices: Timothy Miller, MD, PhD, on the Impact of Tofersen, Antisense Therapeutics in ALS
November 16th 2022The codirector of the ALS Center at Washington University School of Medicine in St. Louis provided perspective on the phase 3 VALOR findings of tofersen, an antisense agent currently under review to treat SOD1-mutated ALS.
Critical Takeaways, Learning Experiences From the Tofersen Trials in ALS: Timothy Miller, MD, PhD
November 11th 2022The codirector of the ALS Center at Washington University School of Medicine provided perspective on the lessons learned from the trials of tofersen, as it aims to become the first FDA-approved therapy specific to SOD1 ALS. [WATCH TIME: 4 minutes]
Role of Tofersen Among Other Previously Approved ALS Therapies: Timothy Miller, MD, PhD
November 10th 2022The codirector of the ALS Center at Washington University School of Medicine in St. Louis provided background on how tofersen would be used alongside other ALS medications if approved. [WATCH TIME: 3 minutes]
Use of Nanoparticles to Combat Neurodegenerative Disease
November 10th 2022Available neurodegenerative disease treatments are generally unsafe and ineffective at penetrating the blood-brain barrier, though the use of nanoparticles can provide improved penetration and exert a neuroprotective effect.
NurOwn Stromal Cell Therapy Continues to Show Positive Data in Less Severe ALS
November 9th 2022After removing participants at higher risk of reaching a floor effect of the ALSFRS-R, those treated with NurOwn demonstrated a higher rate of clinical response and less function lost across 28 weeks than placebo.