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.
May 1st 2025
Moira Kapral, MD, MSc, reflects on her SEQUINS Hall of Fame recognition, the evolution of brain health equity, and her work addressing disparities in stroke care.
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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NeuroVoices: Wayne Feng, MD, FAHA, on Changing the Approach to Poststroke Neuromuscular Symptoms
October 12th 2022The division chief of stroke and vascular neurology at Duke Health discussed how clinicians have typically treated poststroke motor symptoms and how technology is expanding these capabilities.
Approval of AMX0035 and the Future of Clinical Trials for ALS: Lawrence Steinman, MD
October 11th 2022The Zimmermann Professor of Neurology and Neurological Sciences and Pediatrics at Stanford University discussed updates in ALS research along with the need in future studies following AMX005’s (Relyvrio; Amylyx Pharmaceuticals) recent FDA approval. [WATCH TIME: 4 minutes]
Putting Pen to Paper: Executing on Approaches for Neurorestoration
October 11th 2022Joe Kardine, MS, OTR, CBIS, the clinical program manager at the Jefferson Center for Neurorestoration, discussed the influx of new neurorestorative technology and the future of treating patients with functional loss.
Approach to Poststroke Neuromuscular and Motor Symptoms: Wayne Feng, MD, FAHA
October 10th 2022The division chief of stroke and vascular neurology at Duke Health provided insight on the nuances of poststroke neuromuscular symptoms and how they differ from other typically managed impairments. [WATCH TIME: 5 minutes]
Breakthrough in ALS With FDA Approval of AMX0035: Lawrence Steinman, MD
October 10th 2022The Zimmermann Professor of Neurology and Neurological Sciences and Pediatrics at Stanford University discusses Amylyx’s therapy that was recently approved by the FDA, and what it means for the ALS community and the global healthcare industry. [WATCH TIME: 5 minutes]
NeurologyLive® Top Expert Interviews: September 2022
October 6th 2022Expert clinicians offer their perspectives on developmental milestones for children, the NMSOD patient perspective, effective treatments for insomnia, ALS, diabetic and inflammatory neuropathies, and lecanemab in early Alzheimer disease.
Advancing the Treatment Paradigm for Myasthenia Gravis and Looking Toward the Future
October 5th 2022Nicholas Silvestri, MD, clinical professor at the University at Buffalo discussed topics related to the expansion of treatments for myasthenia gravis and the positive outlook of managing the disease.
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.