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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Drs Marcelo Kerstenetzky, Rodrigo de Holanda Mendonça and Adriana Banzzatto Ortega discuss role of primary care physicians and pediatricians in monitoring in SMA. Drs Marcelo Kerstenetzky, Rodrigo de Holanda Mendonça e Adriana Banzzatto Ortega discutem o papel de médicos da atenção primária e pediatras no monitoramento da atrofia muscular espinhal.
Drs Juliana Gurgel Giannetti, Rodrigo de Holanda Mendonça and John Brandsema highlight the importance of multidisciplinary care teams in monitoring and rehabilitation of patients with spinal muscular atrophy. Drs Juliana Gurgel Giannetti, Rodrigo de Holanda Mendonça e John Brandsema enfatizam a importância de equipes multidisciplinares no monitoramento e reabilitação de pacientes com atrofia muscular espinhal.
Using the Myasthenia Gravis Patient Registry Management for Research Studies: Paul Strumph, MD
February 9th 2023The lead of the Myasthenia Gravis Foundation of America’s Global Myasthenia Gravis Patient Registry spoke about the registry from a research project perspective. [WATCH TIME: 4 minutes]
Significance of Relaunched Patient Registry for Myasthenia Gravis Research: Richard Nowak, MD, MS
February 7th 2023The assistant professor of neurology at Yale School of Medicine spoke about the importance of the relaunch of the patient registry for research in myasthenia gravis. [WATCH TIME: 3 minutes]
Sanofi Discontinues Phase 3 Study of Tolebrutinib in Myasthenia Gravis
February 7th 2023The trial was originally placed on hold because of reported cases of drug-induced liver injury in patients who received the study drug, potentially caused by a preexisting factors related to hepatic dysfunction.
National Women Physicians Day: 10 Moments That Matter in Medicine
February 3rd 2023The list of women's contributions to the field of medicine is a long one, encompassing the hard work of hundreds of individuals. To highlight some of these pioneers, NeurologyLive® has compiled a timeline of 10 of the moments that matter in the history of women in medicine.
The expert panel comments on ideal patient profiles for SMA treatment with nusinersen, onasemnogene abeparvovec, and risdiplam. O painel de especialistas comenta os perfis ideais de pacientes para tratamento da atrofia muscular espinhal com nusinersena, onasemnogeno abeparvoveque e risdiplam.
Drs Rodrigo de Holanda Mendonça and Adriana Banzzatto Ortega discuss clinical trial data with nusinersen, onasemnogene abeparvovec, and risdiplam. Drs Rodrigo de Holanda Mendonça e Adriana Banzzatto Ortega discutem dados de testes clínicos com nusinersena, onasemnogeno abeparvoveque e risdiplam.
NeuroVoices: Rachel Alvarez on Identifying Key Treatment Needs for Congenital Muscular Dystrophies
February 1st 2023The executive direct of Cure CMD discussed the state of care for patients with congenital muscular dystrophies, the need for new treatments, and the patience with gene therapies.
Mission Behind Cure CMD’s Research Roundtable, Efforts Around CMDs: Rachel Alvarez
January 28th 2023The executive director of Cure CMD discussed efforts being done to bring the stakeholder community together and improve the care for patients with congenital muscular dystrophies. [WATCH TIME: 4 minutes]
Drs Juliana Gurgel Giannetti and Rodrigo de Holanda Mendonça provide an overview of available treatments for spinal muscular atrophy in Brazil. / Drs Juliana Gurgel Giannetti and Rodrigo de Holanda Mendonça discutem tratamentos para atrofia muscular espinhal disponíveis no Brasil.
Drs Juliana Gurgel Giannetti and Rodrigo de Holanda Mendonça discuss genetic changes in spinal muscular atrophy and genetic screening in Brazil. / Drs Juliana Gurgel Giannetti and Rodrigo de Holanda Mendonça discutem mudanças genéticas em atrofia muscular espinhal e screening genético no Brasil.