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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Unraveling the Complexities of Cerebral Palsy and Building Hope for a Brighter Future
March 24th 2022During Cerebral Palsy Awareness Month, leaders in the field expressed their love for the CP community, the progress made in managing the disorders, and the steps needed to advance care into a realm never before seen.
Gaining a Clearer Picture of Gene Therapy in Neuromuscular Disease: Carsten G. Bönnemann, MD
March 23rd 2022The senior investigator in the Neuromuscular and Neurogenetic Disorders of Childhood Section at NINDS shared his perspective on the data coming out of neuromuscular research and the promise of genetic medicine. [WATCH TIME: 2 minutes]
Subcutaneous Efgartigimod Shows Noninferiority to IV Formulation in Generalized Myasthenia Gravis
March 23rd 2022Topline data from the phase 3 ADAPT-SC study (NCT04735432) suggest that the subcutaneous formulation of the Argenx product was statistically noninferior to the recently approved IV formulation, efgartigimod afla-fcab (Vyvgart).
Improving and Expanding Access to ALS Treatment Options: Calaneet Balas, MSc, MBA
March 23rd 2022The president and chief executive officer of The ALS Association provided commentary on the most pertinent issues with managing patients with ALS and the ways to improve current treatment standards. [WATCH TIME: 4 minutes]
The Need for Participation in Neuromuscular Disease Clinical Trials: Donald S. Wood, PhD
March 22nd 2022The president and CEO of the Muscular Dystrophy Association spoke about the need for increased participation in neuromuscular disorder clinical trials and the rapid therapeutic progress the field is seeing. [WATCH TIME: 4 minutes]
BBP-418 Demonstrates Significant Impact on Root Cause of Limb-Girdle Muscular Dystrophy Type 2i
March 22nd 2022Eleven of the 12 individuals demonstrated at least a 50% reduction in creatine kinase, with 75% of participants reaching twice the normal range, suggesting a reduction in muscle breakdown.
Successes and Challenges in the New Era of Genetic Medicine: Nicholas E. Johnson, MD, MSci
March 20th 2022The division chief of neuromuscular disorders and vice-chair of research at Virginia Commonwealth University spoke to the ongoing success in the development of gene-mediated therapies and the challenges that come along with treating rare neuromuscular disease. [WATCH TIME: 3 minutes]
NeurologyLive® Brain Games: March 20, 2022
March 20th 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 optic neuritis and neuromyelitis optica spectrum disorder (NMOSD).
Zolgensma Reinforces Efficacy in SPR1NT Results of Patients With SMA and 3 Copies of SMN2
March 19th 2022The gene therapy from Novartis showed that it was both efficacious and well-tolerated in presymptomatic patients with SMA with 3 copies of SMN2, with no patients experiencing treatment-related serious adverse events.
Understanding the Newest Therapeutic Advances in Neuromuscular Diseases
March 17th 2022Jinsy Andrews, MD, MSc, FAAN, offered her perspective on the ongoing therapeutic boom in neuromuscular disease and some of the critical needs for physicians to keep up with a rapidly progressing treatment paradigm.
MDA 2022 Highlights the Therapeutic Advances for Neuromuscular Diseases: Donald S. Wood, PhD
March 17th 2022The president and CEO of the Muscular Dystrophy Association spoke about the highlights of this year’s annual meeting and the recent therapeutic advances for neuromuscular disorders. [WATCH TIME: 3 minutes]
Managing New Therapies for Neuromuscular Diseases: Jinsy Andrews, MD, MSc
March 16th 2022The director of neuromuscular clinical trials at Columbia University Irving Medical Center shared her perspective on the challenges of familiarizing oneself with the influx of novel medications for neuromuscular diseases. [WATCH TIME: 3 minutes]
The Revolution in Limb-Girdle Muscular Dystrophy With Gene Therapies: Nicholas E. Johnson, MD, MSci
March 16th 2022The division chief of neuromuscular disorders and vice-chair of research at Virginia Commonwealth University offered his insight into the advances in genetic approaches to LGMD. [WATCH TIME: 2 minutes]
Diagnostic Delays and the Importance of the Physician-Patient Relationship: Amy Shinneman
March 15th 2022The 2022 MDA National Ambassador and patient with Bethlem myopathy spoke to the critical need to improve diagnosis delays in neuromuscular disorders and effective communication between patients and their physicians. [WATCH TIME: 2 minutes]