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.
May 16th 2024
Solid Biosciences is expecting to provide initial safety data on SGT-003 from the first couple of pediatric patients with Duchenne muscular dystrophy enrolled in the phase 1/2 INSPIRE Duchenne trial in mid-2024.
Improving Care for Patients with Motor Complications of Parkinson Disease: Role of Technology and Data
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Clinical Consultations™: Considerations for Customizing Care Plans for Patients with Parkinson Disease Psychosis
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Evolving Perspectives in Alzheimer's Disease: Reaching an Earlier Diagnosis, Understanding Neuroinflammation, and Exploring Therapeutic Advances
September 15, 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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Recognizing Rett Syndrome Early to Improve Long-term Management Outcomes
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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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Burst CME: Optimizing Therapy in Parkinson’s Disease
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Community Practice Connections™: Transforming Multiple Sclerosis Care – Clinical Updates on the Effects of BTK Inhibitors
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Burst Expert Illustrations & Commentary™: Visualizing the Role of the Complement Pathway in Neurological Disorders
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Burst Expert Illustrations & Commentary™ : Visualizing the Role of the Complement Proteins in Neurologic Disorders
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Burst Expert Illustrations & Commentary™: Visualizing the Implications of Anti-Complement Therapies on Generalized Myasthenia Gravis
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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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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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Pridopidine’s Impact on ALS, Assessing Results From HEALEY ALS Platform Trial: Melanie Leitner, PhD
May 23rd 2023The consultant and founder of Accelerating NeuroVentures talked about the pridopidine arm from the HEALEY ALS Platform trial, which showed significant benefits in speech measures. [WATCH TIME: 5 minutes]
Investigating Losmapimod in Phase 2 Trial for Facioscapulohumeral Muscular Dystrophy
May 15th 2023Leo H. Wang, MD, PhD, FAAN, associate professor of neurology, University of Washington Medical Center, talked about the phase 2 study of losmapimod for facioscapulohumeral muscular dystrophy presented at the 2023 AAN Annual Meeting.
FDA Approves Enzyme Replacement Therapy Pegunigalsidase Alfa for Fabry Disease
May 10th 2023To date, the enzyme replacement therapy had been studied in a comprehensive clinical development program that comprised more than 140 patients with Fabry disease with up to 7.5 years of follow up treatment.
AOC 1001 as a Potential Treatment for Myotonic Dystrophy Type 1
May 9th 2023Nicholas Johnson, MD, MSci, FAAN, division chief of neuromuscular disorders and vice chair of research, Virginia Commonwealth University, provided insight on promising phase 1/2 data of AOC 1001 in myotonic dystrophy type 1.
Evolving Landscape of Pediatric Neurology: Erika Fullwood Augustine, MD, MS
May 5th 2023The associate chief science officer at Kennedy Krieger Institute shared thoughts on the state of pediatric neurology and the significance of being named winner of the Sydney Carter Award. [WATCH TIME: 3 minutes]
AAN Annual Meeting 2023: Top Expert Interviews
May 2nd 2023A group of experts in the care of patients with neurological conditions—Ali Jannati, MD, PhD; Madhav R. Thambisetty, MD, PhD; Daniel Ontaneda, MD, PhD; Peter J. Goadsby, MD, PhD, DSc; Andrew Russman, DO—shared their perspectives on hot topics of treatment and management in neurology from the 2023 American Academy of Neurology Annual Meeting.
Serial Assessments Used in Groundbreaking Brain Study: Imad Najm, MD
May 2nd 2023The director of Cleveland Clinic’s Epilepsy Center at the Cleveland Clinic Neurological Institute provided perspective on the in-depth testing neurologically healthy patients in the Brain Study will undergo. [WATCH TIME: 3 minutes]