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 10th 2024
Avidity Biosciences is anticipated to initiate its phase 3 HARBOR trial assessing AOC 1001 as a treatment for patients with myotonic dystrophy type 1 in the second quarter of 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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Global Impacts of Rare Disease Day for Patients With Neurologic Disorders: Jill Morris, PhD
February 29th 2024The program director in the division of Neuroscience at the National Institute of Neurological Disorders and Stroke provided context on the importance of Rare Disease Day and strides made over the years. [WATCH TIME: 5 minutes]
Advocacy and Progress in Rare Neuromuscular Diseases From a Policy Standpoint: Paul Melmeyer, MPP
February 29th 2024In honor of Rare Disease Day, the vice president of public policy and advocacy at the Muscular Dystrophy Association shared his perspective of advocacy and policy progress in the realm of rare neuromuscular diseases. [WATCH TIME: 6 minutes]
Preclinical Studies of ATH-1105 Highlight Therapeutic Potential in ALS
February 27th 2024ATH-1105 protected various cell culture systems from glutamate-mediated toxicity and its pathological alterations, which include mitochondrial dysfunction, apoptotic signaling, and TDP-43 mis-localization, among others.
Future Role of Neurofilament Light in ALS Drug Development, Clinical Trials: Rita Sattler, PhD, MSc
February 23rd 2024The professor in the Department of Translational Neuroscience at Barrow Neurological Institute provided perspective on the approval of tofersen (Qalsody; Biogen) for patients with ALS and the ways neurofilament light will be used going forward. [WATCH TIME: 3 minutes]
Highlighting Genetic Discoveries and Therapeutic Developments in ALS: Matthew B. Harms, MD
February 21st 2024The associate professor of neurology at Columbia University talked about a track session that he will be chairing at the upcoming MDA conference on amyotrophic lateral sclerosis genetic research. [WATCH TIME: 5 minutes]
NeuroVoices: Rita Sattler, MD, MSc, on 2024 MDA Conference and Advances in ALS Research
February 21st 2024The professor in the Department of Translational Neuroscience at Barrow Neurological Institute discussed several topics related to ALS research and the emerging biomarkers in recent years ahead of the 2024 MDA Clinical and Scientific Conference.
The Emergence of Novel Biomarkers and Model Systems for ALS: Rita Sattler, MD, MSc
February 19th 2024The professor in the Department of Translational Neuroscience at Barrow Neurological Institute provided comments on her session track at the upcoming 2024 MDA conference and some of the recent advancements in ALS research. [WATCH TIME: 4 minutes]
European Commission Approves Omaveloxolone as First Therapy for Friedreich Ataxia
February 14th 2024Omaveloxolone is an oral, once-daily medication indicated for the treatment of Friedreich’s ataxia in adults and adolescents aged 16 years and older in both the United States and the European Union.