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 19th 2025
Backed by promising early data and mechanistic insights, SNUG01 enters clinical trials as the first TRIM72-based gene therapy candidate for ALS.
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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Endoplasmic Reticulum and/or Mitochondrial-Dependent Neuronal Degeneration in ALS
June 14th 2022Given the importance of mitochondrial and endoplasmic reticulum function in ALS, the disruption of intracellular mitochondria-endoplasmic reticulum contacts presents yet another avenue for neuronal degeneration—perhaps the primary point of underlying dysfunction.
FibroGen Completes Enrollment in LELANTOS-2 Trial of Pamrevlumab in Ambulatory DMD
June 9th 2022The trial is set to read out topline data in the second half of 2023 after completing the target enrollment of 73 patients with Duchenne muscular dystrophy aged 6 to 12 years. The connective tissue growth factor inhibitor has been granted orphan drug, fast track, and rare pediatric disease designations.
Clinical Care Effects of Risdiplam’s Expanded Indication in SMA
June 8th 2022Richard Finkel, MD, RAINBOWFISH’s principal investigator and the director of the Experimental Neuroscience Program at St. Jude Children’s Research Hospital, offered his take on the recent FDA decision for risdiplam (Evrysdi; Genentech).
NeuroVoices: Michael Sughrue, MD, on Using Machine Learning to Build a Better Brain Map
June 1st 2022The founder and chief medical officer of Omniscient Neurotechnology discussed how the use of machine learning and big data will be critical in revolutionizing our understanding of the brain and mental illnesses.
Solving the Biggest Questions About the Brain With Advanced Technology: Michael Sughrue, MD
May 31st 2022The founder and chief medical officer of Omniscient Neurotechnology commented on the areas of need with understanding the brain map and treating conditions with multiple pathway crossover. [WATCH TIME: 4 minutes]
Phase 2 Data Show ACE-083 Was Safe, Improved Muscle Function in Charcot-Marie-Tooth Disease Type 1
May 22nd 2022Despite good tolerability with few drug-related treatment emergent AEs and increased muscle volume, the failure to provide clinically relevant improvements in muscle function led to the discontinuation of ACE-083 for CMT in 2020.