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
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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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Limits, Cautions When Using NuroSleeve to Restore Arm Function: Joe Kardine, MS, OTR, CBIS
October 4th 2022The clinical program manager at the Jefferson Center for Neurorestoration discussed certain scenarios in which the NuroSleeve system may not be applicable for patients with neurological disorders. [WATCH TIME: 3 minutes]
CNM-Au8 Shows Survival Benefit in HEALEY ALS Trial, But Fails to Meet End Points
October 3rd 2022Despite not meeting its primary and secondary end points, CNM-Au8 will continue to be evaluated in an open-label extension in HEALEY ALS and could potentially be offered in an expanded access protocol program.
Progression of New Treatments for Duchenne Muscular Dystrophy: Emma Ciafaloni, MD
October 3rd 2022The professor of Neurology and Pediatrics at University of Rochester Medical Center discusses the progression of new treatment for Duchenne muscular dystrophy and provides suggestions for moving forward with research in the field. [WATCH TIME: 5 minutes]
Recommendations for Clinical Trial Design in Duchenne Muscular Dystrophy: Emma Ciafaloni, MD
October 2nd 2022The professor of neurology and pediatrics at University of Rochester Medical Center gives some recommendations for the improvement in clinical trial design for patients with rare diseases such as Duchenne muscular dystrophy. [WATCH TIME: 5 minutes]
NeurologyLive® Brain Games: October 2, 2022
October 2nd 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 general neurology, related to the recent 4th Annual International Congress on the Future of Neurology®.
Reactions, Ramifications, and Forward Progress of Newly Approved AMX0035 for ALS
October 1st 2022Justin Klee and Josh Cohen, cofounders and cochief operating officers, Amylyx Pharmaceuticals, answered questions related to the new approval of AMX0035 for ALS and how it changes the treatment landscape going forward.
Sleep Breathing Disorders Appear Common for Patients with Duchenne Muscular Dystrophy
September 30th 2022A questionnaire-based study from Saudi Arabia on sleep quality revealed that sleep-related disorders, such as obstructive sleep apnea, are relatively common in patients with Duchenne muscular dystrophy.
Open-Label Extension Data Highlight Tofersen’s Ability to Reduce SOD1 Protein in ALS
September 29th 2022Despite not improving clinical end points, treatment with tofersen was associated with reduced in the total concentration of SOD1 protein and neurofilament light chain, a marker of axonal injury.
Diagnosis of Myotonic Dystrophy Demonstrates an Association With Depression
September 29th 2022Findings from a cross-sectional survey revealed a significant association between depression and a diagnosis of myotonic dystrophy, and that symptoms impacting quality of life are more common in neuromuscular disorders.
New Treatments for Duchenne Muscular Dystrophy: Emma Ciafaloni, MD
September 27th 2022New Treatments for Duchenne Muscular Dystrophy: Emma Ciafaloni, MD The professor of Neurology and Pediatrics at University of Rochester Medical Center discusses the new and future treatments for Duchenne muscular dystrophy. [WATCH TIME: 4 minutes]
Tele-Yoga Breathing for Respiratory Function Proves Feasibility in Amyotrophic Lateral Sclerosis
September 26th 2022A nonrandomized pilot study demonstrated high adherence rates and acceptability ratings with tele-yoga breathing in patients with ALS, proving that it is a safe and feasible practice.
Patients Receiving Efgartigimod Respond to COVID-19 Vaccination With IgG Antibodies
September 25th 2022In an investigation on the effect of treatment with efgartigimod on humoral immune responses to COVID-19 vaccination, the immunization resulted in antigen-specific IgG responses in most patients.