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.

Latest News

5-Year Outcomes Add Supportive Evidence to Approved Gene Therapy for Duchenne
5-Year Outcomes Add Supportive Evidence to Approved Gene Therapy for Duchenne

October 8th 2024

No serious adverse events were reported, and the therapy demonstrated a favorable safety profile over the 5-year study period.

Ari Azhir, PhD  (Credit: LinkedIn)
Neuvivo Submits NDA to FDA for Investigational ALS Treatment NP001

October 8th 2024

PTC to Submit NDA for Vatiquinone in Friedreich Ataxia Following Positive Long-Term Extension Data
PTC to Submit NDA for Vatiquinone in Friedreich Ataxia Following Positive Long-Term Extension Data

October 8th 2024

FDA Grants Orphan Drug Designation to Becker and Duchenne Agent RAG-18
FDA Grants Orphan Drug Designation to Becker and Duchenne Agent RAG-18

October 8th 2024

Muscle-Directed Therapy Apitegromab Meets Primary End Point in Phase 3 SAPPHIRE Trial of Spinal Muscular Atrophy
Muscle-Directed Therapy Apitegromab Meets Primary End Point in Phase 3 SAPPHIRE Trial of Spinal Muscular Atrophy

October 7th 2024

Latest CME Events & Activities

Cases and Conversations™: Applying Best Practices to Prevent Shingles in Your Practice

October 16, 2024

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Mastering MS: Translating Evidence into Optimal Management Plans

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Advances In Treating Migraine in Your OB/GYN Practice: Navigating Treatment Paradigms to Improve Patient Care

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Patient, Provider, and Caregiver Connection™: Pediatric Myasthenia Gravis - Current Treatment and Emerging Con...

November 12, 2024

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Recognizing Rett Syndrome Early to Improve Long-term Management Outcomes

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2024 Neuromuscular Summit

November 20, 2024

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5th Annual International Congress on the Future of Neurology®

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Clinical Consultations™: Managing Depressive Episodes in Patients with Bipolar Disorder Type II

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Advances In™ Generalized Myasthenia Gravis: Improving Patient Outcomes Through Early Diagnosis and Management

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Medical Crossfire®: Understanding the Advances in Bipolar Disease Treatment—A Comprehensive Look at Treatment Selection Strategies

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'REEL’ Time Patient Counseling: The Diagnostic and Treatment Journey for Patients With Bipolar Disorder Type II – From Primary to Specialty Care

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Community Practice Connections™: Transforming Multiple Sclerosis Care – Clinical Updates on the Effects of BTK Inhibitors

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Burst CME: Optimizing Therapy in Parkinson’s Disease

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Burst Expert Illustrations & Commentary™ : Visualizing the Role of the Complement Proteins in Neurologic Disorders

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Addressing Healthcare Inequities: Bridging the Gap in Multiple Sclerosis – A Focus on Clinical and Healthcare Disparities in Black Patients

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Burst Expert Illustrations & Commentary™: Visualizing the Implications of Anti-Complement Therapies on Generalized Myasthenia Gravis

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Burst Expert Illustrations & Commentary™: Visualizing the Role of the Complement Pathway in Neurological Disorders

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Individualizing Treatment for Patients with Generalized Myasthenia Gravis

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Patient, Provider & Caregiver Connection™: Reducing the Burden of Parkinson Disease Psychosis with Personalized Management Plans

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Clinical ShowCase™ in ALS: Addressing Diagnostic Delays, Evolving Therapies, and Multidisciplinary Care

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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 & Commentary™: Visualizing the Role of FcRN in Neurological Disorders

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BURST Expert Illustrations and Commentaries™: Visualizing the Implications of FcRN-Targeted Therapies on Generalized Myasthenia Gravis

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SimulatED™: Diagnosing and Treating Alzheimer’s Disease in the Modern Era

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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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Clinical Consultations™: Navigating the Evolving Treatment Landscape in Generalized Myasthenia Gravis

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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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