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 8th 2025
Newly presented open-label extension results at AAN 2025 suggest that investigational ZYN002, a cannabidiol gel, may help improve irritability in children and adolescents living with Fragile X syndrome.
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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Identifying and Treating CIDP in the Modern Era
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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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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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Neurologist’s Role in Addressing Healthcare Disparities: A. Gordon Smith, MD, FAAN
January 7th 2024The professor and chair of neurology at Virginia Commonwealth University discussed how neurologists are urged to take organized action and provide training on healthcare disparities, with a particular focus on race, ethnicity, and financial barriers to access. [WATCH TIME: 2 minutes]
Dyne Reports Positive Phase 1/2 Data for Duchenne Agent DYNE-251
January 6th 2024After 6 months of treatment, once every 4-week administration of DYNE-251 reached levels of dystrophin expression, exon skipping, and percent dystrophin positive fibers that exceeded levels reported in a previous trial of eteplirsen, considered the standard of care.
Steps to Validate Stathmin-2 as an ALS-Specific Biomarker: Cathleen Lutz, PhD
January 4th 2024The vice president of the Rare Disease Translational Center at the Jackson Laboratory provided commentary on the next steps needed to further understand stathmin-2’s role as a potential disease-modifying biomarker. [WATCH TIME: 3 minutes]
Clinical Potential of AOC 1044 in Treating Duchenne Muscular Dystrophy Amenable to Exon 44 Skipping
January 2nd 2024Steve Hughes, MD, chief medical officer at Avidity Biosciences, provided commentary on recently announced positive topline data for AOC 1044, an investigational agent for Duchenne muscular dystrophy.
Episode 105: Recapping Conversations in Neurology From 2023
December 29th 2023Mind Moments®, a podcast from NeurologyLive®, brings you a review of 2023, with insights from Sharon Cohen, MD, FRCPC; Robert A. Hauser, MD, MBA; Alberto Espay, MD, PhD; Carolina Ferreira Atuesta, MD, MSc; and Natalie Goedeker, CPNP. [LISTEN TIME: minutes]
Rett Syndrome Symptoms and Quality of Life Improvements Through Trofinetide: Ponni Subbiah, MD, MPH
December 21st 2023The chief medical officer of Acadia Pharmaceuticals provided commentary on a caregiver analysis assessing beneficial experiences with trofinetide in patients with Rett syndrome. [WATCH TIME: 4 minutes]
NeuroVoices: Ponni Subbiah, MD, MPH, on the Open-Label LILAC-2 Study of Trofinetide
December 20th 2023The senior vice president, global head of medical affairs, and chief medical officer at Acadia Pharmaceutical, gave an overview of LILAC-2, a long-term extension study assessing trofinetide (Daybue), the first approved therapy for Rett syndrome.