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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Advocacy and Empowerment: Finding Your Advocacy Community
November 9th 2022The Palatucci Advocacy Leadership Forum, or PALF, sponsored by the American Academy of Neurology, gives neurologists and trainees tools to successfully advocate for their ideas and develop their identity as physician advocates.
Understanding Tofersen’s Impact on SOD1 ALS, Neurofilament Light: Timothy Miller, MD, PhD
November 9th 2022The codirector of the ALS Center at Washington University School of Medicine in St. Louis discussed the long-term data of VALOR and its open-label extension assessing tofersen (Biogen) in SOD1 ALS. [WATCH TIME: 4 minutes]
Patient-Reported Outcomes Worsen as ALS Disease Progresses, Real-World Study Shows
November 4th 2022On King’s staging systems, ALSAQ-5 scores increased from 24.6 at stage 1 to 62.1 at stage 4, whereas for Milano-Torino Staging systems, patients’ ALSAQ-5 scores increased from 43.2 at stage 0 to 80.7 at stage 3.
Palatability Strategies Show Promise for Addressing AMX0035 Taste Concerns Among Patients With ALS
November 2nd 2022As patients with ALS may have altered taste perception and have expressed that bitterness is associated with AMX0035 (Relyvrio; Amylyx) administration, a number of coping strategies have suggested a workaround to ensure treatment adherence and effectiveness.
The ALS MOTOR as a Beneficial and Accessible Tool for Virtually Tracking ALS Progression
November 2nd 2022Christina Fournier, MD, MSc, associate professor of neurology, Emory University, discussed her presentation at the 2022 NEALS Consortium using a telemedicine exam to track ALS progression.
SBT-272 Shows Neuroprotective Impacts in ALS Mouse Models of TDP-43 Pathology
November 2nd 2022SBT-272, an investigational small molecule in development for ALS and other neurological disease of mitochondrial dysfunction, resulted in improved membrane potential and axonal outgrowth of TDP-43 in vitro.
Muscle-Directed Therapy Apitegromab Improves Quality of Life in Spinal Muscular Atrophy
October 25th 2022Over a 24-month treatment period, apitegromab-treated patients showed improvements on PEDI-CAT and PROMIS, measures of activities of daily living, and ESBBT, a muscle endurance measurement tool.
NeurologyLive® Brain Games: October 23, 2022
October 23rd 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 European Committee for Treatment and Research in Multiple Sclerosis.