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 17th 2025
The chief scientific officer at Avidity Biosciences details the mechanism, early results, and future plans for Del-zota, an investigational therapy targeting exon 44 in Duchenne muscular dystrophy.
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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Assessing the Therapeutic Potential of AOC 1001 in Myotonic Dystrophy Type 1
November 11th 2023Steve Hughes, MD, chief medical officer at Avidity, provided perspective on recently announced positive phase 1/2 and OLE data evaluating AOC 1001, an investigational antisense oligonucleotide, in patients with myotonic dystrophy type 1.
Targeting Neurotrophic Factors and Preclinical Results of ALS Agent ATH-11055: Kevin Church, PhD
November 8th 2023The chief scientific officer at Athira Pharma talked about new preclinical results presented at the 2023 NEALS Annual Meeting on the investigational therapy ATH-1105 for patients with amyotrophic lateral sclerosis. [WATCH TIME: 5 minutes]
Inclusive ALS Grant Expands Access to CNM-Au8 Therapy for ALS: Karen S. Ho, PhD
November 8th 2023The vice president of translational medicine at Cene Nanomedicine talked about a recently funded grant that is focused on inclusivity and the collection of real-world data on the efficacy of CNM-Au8 in various stages of amyotrophic lateral sclerosis. [WATCH TIME: 7 minutes]
Neurologists' Perspectives on Healthcare Disparities in Myasthenia Gravis: A. Gordon Smith, MD, FAAN
November 7th 2023The professor and chair of neurology at Virginia Commonwealth University discussed a study conducted among neurologists that revealed healthcare disparities in the treatment of myasthenia gravis, particularly related to access to care and therapeutics. [WATCH TIME: 5 minutes]
Study Reveals Racial Disparities in Acute Care Utilization Among Patients With Myasthenia Gravis
November 7th 2023Recent research, presented at the 2023 AANEM Annual Meeting, emphasized the potential need for patient support programs targeting at-risk populations to enhance disease management and reduce acute care utilization in myasthenia gravis.
How Clinicians can Advocate for Patients Beyond the Clinic: Michael Lewis, MA
November 7th 2023The director of disability policy at the Muscular Dystrophy Association discussed his lecture at AANEM 2023 on how clinicians can advocate for their patients to help improve accessibility and health equity. [WATCH TIME: 7 minutes]
Majority of Patients With Generalized Myasthenia Gravis Fail to Meet Clinical Trial Criteria
November 7th 2023Among 67 patients with generalized myasthenia gravis studied from January 2021 to June 2023, 89.5% failed to meet clinical trial inclusion criteria, with the most common reason being a Myasthenia Gravis Activities of Daily Living score of less than 5.
Zilucoplan Shows Reduction in MG Worsening in Phase 3 RAISE Study for Generalized Myasthenia Gravis
November 3rd 2023A post hoc analysis of the phase 3 RAISE study evaluating zilucoplan, a complement C5 inhibitor, showed significantly fewer patients experienced worsening and fewer requiring rescue therapy while on medication compared with placebo.
Social Determinants of Health Lead to Greater Suboptimal Treatment Response in Myasthenia Gravis
November 2nd 2023A retrospective study presented at the 2023 AANEM meeting revealed that social determinants of health are linked to poorer treatment outcomes in myasthenia gravis patients, emphasizing the need for identifying and supporting at-risk individuals.
Vutrisiran Shows Benefit Across Baseline Severities in Phase 3 HELIOS-A Study
November 2nd 2023In a recent post hoc analysis presented at the AANEM meeting, findings highlight the positive impact of vutrisiran on quality of life, disability, gait speed, and nutritional status across different neuropathy impairment score quartiles.