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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Future Role of Neurofilament Light in ALS Drug Development, Clinical Trials: Rita Sattler, PhD, MSc
February 23rd 2024The professor in the Department of Translational Neuroscience at Barrow Neurological Institute provided perspective on the approval of tofersen (Qalsody; Biogen) for patients with ALS and the ways neurofilament light will be used going forward. [WATCH TIME: 3 minutes]
Highlighting Genetic Discoveries and Therapeutic Developments in ALS: Matthew B. Harms, MD
February 21st 2024The associate professor of neurology at Columbia University talked about a track session that he will be chairing at the upcoming MDA conference on amyotrophic lateral sclerosis genetic research. [WATCH TIME: 5 minutes]
NeuroVoices: Rita Sattler, MD, MSc, on 2024 MDA Conference and Advances in ALS Research
February 21st 2024The professor in the Department of Translational Neuroscience at Barrow Neurological Institute discussed several topics related to ALS research and the emerging biomarkers in recent years ahead of the 2024 MDA Clinical and Scientific Conference.
The Emergence of Novel Biomarkers and Model Systems for ALS: Rita Sattler, MD, MSc
February 19th 2024The professor in the Department of Translational Neuroscience at Barrow Neurological Institute provided comments on her session track at the upcoming 2024 MDA conference and some of the recent advancements in ALS research. [WATCH TIME: 4 minutes]
European Commission Approves Omaveloxolone as First Therapy for Friedreich Ataxia
February 14th 2024Omaveloxolone is an oral, once-daily medication indicated for the treatment of Friedreich’s ataxia in adults and adolescents aged 16 years and older in both the United States and the European Union.
Reassessing the Process of Drug Development for Duchenne Muscular Dystrophy: Dongzhe Hong, PhD
February 13th 2024The postdoctoral research fellow at Brigham and Women’s Hospital and Harvard Medical School discussed some of the issues with ensuring timely care access to approved therapies for patients with Duchenne muscular dystrophy. [WATCH TIME: 3 minutes]
Advancements and Challenges in Genetic Treatments for Neuromuscular Diseases: John W. Day, MD, PhD
February 12th 2024Ahead of the 2024 MDA Conference, the medical advisor and care center director at Muscular Dystrophy Association talked about the track sessions he will be cochairing at the meeting. [WATCH TIME: 5 minutes]
NeurologyLive® Brain Games: February 11, 2024
February 11th 2024Test 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 neuromuscular disorders, and specifically, Duchenne muscular dystrophy.
NeuroVoices: Joe Burnett, on FDA Clearance of SmartFrame OR Stereotactic System
February 7th 2024The chief executive officer of ClearPoint Neuro provided clarity on how the recently approved SmartFrame system can enhance the efficiency of operating room procedures, allowing for flexible workflows and image-based corrections.