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

Rare Disease Stories of Success: Newfound Hope for Spinal Muscular Atrophy
Rare Disease Stories of Success: Newfound Hope for Spinal Muscular Atrophy

February 28th 2025

Mary Schroth, MD, FAAP, FCCP, chief medical officer at Cure SMA, shed insight on the advances and promise for treating spinal muscular atrophy, in the context of Rare Disease Day.

NeuroVoices: John Brandsema, MD, on Updates in Gene Therapy at MDA 2025
NeuroVoices: John Brandsema, MD, on Updates in Gene Therapy at MDA 2025

February 26th 2025

Johanna Hamel, MD  (Credit: University of Rochester Medical Center)
Early Phase 1 Trial Data Suggests Therapeutic Potential for PGN-EDODM1 in Myotonic Dystrophy Type 1

February 25th 2025

Dipal Doshi  (Credit: LinkedIn)
FDA Removes Clinical Hold on Duchenne Muscular Dystrophy Agent ENTR-601-44

February 24th 2025

NeurologyLive® Friday 5 — February 21, 2025
NeurologyLive® Friday 5 — February 21, 2025

February 21st 2025

Latest CME Events & Activities

A New Era in NMOSD Treatment: Optimizing Therapeutic Transitions and Reducing Patient Burden

March 1, 2025

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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 & 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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