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
Lewis B. Morgenstern, MD, reflects on his SEQUINS Hall of Fame recognition, the importance of health equity research, and his ongoing efforts to reduce stroke disparities.
Burst Expert Illustrations & Commentary™: Visualizing the Role of FcRN in Neurological Disorders
View More
BURST Expert Illustrations and Commentaries™: Visualizing FcRn as a Therapeutic Target in Neurological Disease
View More
BURST Expert Illustrations and Commentaries™: Visualizing the Implications of FcRN-Targeted Therapies on Generalized Myasthenia Gravis
View More
Clinical Consultations™: Navigating the Evolving Treatment Landscape in Generalized Myasthenia Gravis
View More
Burst Expert Illustrations & Commentary™: Visualizing the Role of Subcutaneous Infusion as an Alternate Administration Route for Medical Interventions
View More
SimulatED™: Diagnosing and Treating Alzheimer’s Disease in the Modern Era
View More
SimulatED™: Understanding the Role of Genetic Testing in Patient Selection for Anti-Amyloid Therapy
View More
Expert Illustrations & Commentaries™: New Targets for Treatment in Cognitive Impairment in Schizophrenia – The Role of NMDA Receptors and Co-agonists
View More
BURST CME™ Part I: Understanding the Impact of Huntington’s Disease
View More
Burst CME™ Part II: The Evolving Treatment Landscape for Huntington Disease
View More
Carolina Neuromuscular Disease Summit
September 27, 2025
Register Now!
SimulatEd™ From Discomfort to Relief: Acute Pain Management Essentials
View More
Evolving Perspectives in Alzheimer's Disease: Reaching an Earlier Diagnosis, Understanding Neuroinflammation, and Exploring Therapeutic Advances
View More
Clinical ShowCase: Developing a Personalized Treatment Plan for a Patient with Huntington’s Disease Associated Chorea
View More
Medical Crossfire®: Integrating Real-World Data to Improve Outcomes for Patients With Multiple Sclerosis
View More
Medical Crossfire® - Optimizing Management for Patients With Generalized Myasthenia Gravis: Focus on Complement Inhibitors
View More
Burst CME™: Setting the Stage – Individualizing Migraine Care for Diverse Populations Across Care Settings
View More
Burst CME™: The Patient Journey – Unmet Needs From Diagnosis Through Management of Migraine
View More
Evolving Perspectives in Alzheimer Disease : Reaching an Earlier Diagnosis, Understanding Neuroinflammation, and Exploring Therapeutic Advances
View More
Burst CME™: Optimizing the Use of CGRP Targeted Agents for the Treatment of Migraine
View More
Burst CME™: Optimizing Migraine Management – Addressing Unmet Needs, Individualizing Care for Diverse Populations, and Utilizing CGRP Targeted Agents
View More
Community Practice Connections™: Optimizing the Management of Tardive Dyskinesia—Addressing the Complexity of Care With Targeted Treatment
View More
Cases and Conversations™: Applying Best Practices to Prevent Shingles in Your Practice
View More
Patient, Provider, and Caregiver Connection™: Pediatric Myasthenia Gravis—Current Treatment and Emerging Concepts
View More
Mind Moments™: Optimizing Management for Patients with Generalized Myasthenia Gravis
View More
Cases and Conversations™: Navigating the Complexities of Managing Myasthenia Gravis in Pediatric and Pregnant Patient Populations
View More
Cases and Conversations™: Little Voices, Big Challenges – Comprehensive Care for Pediatric Spinal Muscular Atrophy
View More
Identifying and Treating CIDP in the Modern Era
View More
A Breath of Strength: Managing Cancer Associated LEMS and Lung Cancer as One
View More
Striking the Right Nerve: Managing Cancer Associated LEMS in Lung Cancer Patients
View More
Restoring Dystrophin Protein Production in DMD With Exon Skipping AOC Therapy: Steve Hughes, MD
September 5th 2023The chief medical officer at Avidity Biosciences talked about how its exon skipping AOC therapies are designed to address the lack of functional dystrophin protein among patients with Duchenne muscular dystrophy to improve muscle function. [WATCH TIME: 4 minutes]
ALS Agent CNM-Au8 Displays Survival Benefit and Delay of Clinical Worsening in 2 Year Follow-Up
September 2nd 2023Findings from an open-label extension trial among 45 patients with ALS showed a significant median survival benefit of 19.3 months and a significant 52% decreased risk of ALS clinical worsening events after treatment.
Creating Efficient, Well-Rounded Neurology Departments
August 31st 2023A panel of former AUPN presidents detailed how advances in neurology have changed the way the clinical specialty is taught, and why collaboration among leadership remains vital to creating an effective neurology department. [WATCH TIME: 7 minutes]
Duchenne Agent Pamrevlumab Fails to Meet Primary End Point in Phase 3 LELANTOS-2 Trial
August 31st 2023Despite pamrevlumab being generally safe and well tolerated, the potential first-in-class connective tissue growth factor inhibitor antibody did not meet its primary endpoint in a phase 3 trial for ambulator Duchenne muscular dystrophy.
Nusinersen Exhibits Long-Term Safety and Efficacy Across Wide Spectrum of SMA Severity
August 31st 2023Findings from a real-world study among 120 patients with SMA suggests that nusinersen administration is safe and well tolerated, with only 30% of the patients mostly experiencing mild adverse events.
EXPLORE44's Approach to Rare Disease Treatment for Duchenne Muscular Dystrophy: Steve Hughes, MD
August 30th 2023The chief medical officer at Avidity Biosciences discussed the phase 1/2 EXPLORE44 trial, assessing an investigational treatment for Duchenne muscular dystrophy, and highlighted the potential of the treatment based on the recent FDA orphan drug and fast track designations. [WATCH TIME: 4 minutes]
NeurologyLive® Clinician of the Month Spotlight: Gary Hisch, MD
August 28th 2023As part of our monthly clinician spotlight, NeurologyLive® highlighted expert Gary Hisch, MD, pediatric neurologist at Cleveland Clinic, who oversees programs for neuromuscular disorders such as for patients with spinal muscular atrophy.