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.
January 30th 2025
The initial cohort of the EPISOD1 trial demonstrated no significant safety concerns for AMT-162, enabling progression to the next dose-escalation phase.
Individualizing Treatment for Patients with Generalized Myasthenia Gravis
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A New Era in NMOSD Treatment: Optimizing Therapeutic Transitions and Reducing Patient Burden
March 1, 2025
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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 & 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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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™: 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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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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Medical Crossfire®: Integrating Real-World Data to Improve Outcomes for Patients With Multiple Sclerosis
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Burst CME™: The Patient Journey – Unmet Needs From Diagnosis Through Management of Migraine
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Burst CME™: Setting the Stage – Individualizing Migraine Care for Diverse Populations Across Care Settings
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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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Japan's Ministry of Health, Labour and Welfare Grants Regulatory Approval to Efgartigimod for CIDP
January 28th 2025Previously, Argenx's efgartigimod alfa and hyaluronidase-qvfc was granted orphan drug designation for the treatment of chronic inflammatory demyelinating polyneuropathy in Japan.
FDA Accepts sNDA for Higher Dosing Regimen of Nusinersen for Spinal Muscular Atrophy
January 23rd 2025In the phase 2/3 DEVOTE study, a higher dose of nusinersen met its primary end point at 6 months, achieving a statistically significant improvement in motor function among treatment-naïve symptomatic infants with SMA.
NeuroVoices: Nigel Bunnett, PhD, BSc, on Identifying a Novel Therapeutic Target for Chronic Pain
January 15th 2025The professor and chair of the Department of Molecular Pathobiology at NYU College of Dentistry discussed groundbreaking research on chronic pain, focusing on the discovery of new mechanisms involving nerve growth factor and its receptors.
FDA Clears IND for Trial Assessing Gene Therapy SGT-212 in Friedreich Ataxia
January 8th 2025SGT-212 delivers full-length frataxin via MRI-guided intradentate nucleus infusion and intravenous administration to target mitochondrial dysfunction in neurons and cardiomyocytes, addressing both neurologic and cardiac symptoms.
Expanding the Clinical Care of Friedreich Ataxia Through Omaveloxlone and SKYCLARYS PASS Registry
January 4th 2025David Lynch, MD, PhD, a professor of neurology at the University of Pennsylvania Perelman School of Medicine, discussed the significance of a number of recently presented analyses highlighting omaveloxlone, the first approved therapy for Friedreich ataxia.