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
Register Now!
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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FDA Approves Insightec’s Exablate Neuro to Treat Second Side of Essential Tremor
December 21st 2022Using focused ultrasound waves, the Exablate Neuro platform has now gained the FDA greenlight for medication-refractory essential tremor, tremor-dominant Parkinson disease, and second side of essential tremor.
Myotonic Dystrophy Agent AOC 1001 Demonstrates Successful Targeted Delivery of RNA
December 15th 2022AOC 1001, an agent consisting of a proprietary monoclonal antibody that binds to the transferrin receptor 1, was safe, tolerable, and showed significant reductions in DMPK, a disease-related mRNA.
Building the Biomarker Panel in Neurology: Glial Fibrillary Acidic Protein
December 9th 2022As the treatment paradigm for neurologic diseases rapidly progresses, the need for more thorough biomarker tools to measure disease progression and severity has increased, and in recent years, GFAP has emerged as a valuable candidate to add to the existing panel.
DNL343 to be Assessed in HEALEY ALS Platform Trial Following Positive Phase 1 Findings
December 6th 2022After showing robust inhibition of biomarkers associated with integrated stress response, the eIF28 modulator will be assessed alongside several other potential agents in the HEALEY ALS Platform trial.
The Expectations for the 2023 MDA Conference: Donald S. Wood, PhD; Sharon Hesterlee, PhD
December 1st 2022The president and CEO, as well as the executive vice president and chief research officer, of MDA, together offered an in-depth overview of what to expect from the 2023 MDA Conference, which will be held from March 19-22, 2023, in Dallas, Texas. [WATCH TIME: 12 minutes]
Expanding the Capabilities of Treating Neuromuscular Poststroke Symptoms: Wayne Feng, MD, FAHA
November 29th 2022The division chief of stroke and vascular neurology at Duke Health provided perspective on the multidisciplinary team needed to treat neuromuscular poststroke symptoms, as well as exciting innovations on the horizon. [WATCH TIME: 4 minutes]
Expanding Neuropalliative Care to New Disease States: Benzi Kluger, MD, MS
November 28th 2022The president of the International Neuropalliative Care Society detailed potential disease states that could benefit from neuropalliative care as well as the importance of the patient community in expanding this type of approach. [WATCH TIME: 4 minutes]