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.
November 22nd 2024
The multicenter, 24-week study will feature 20 patients with MG, testing changes on MG-ADL as well as several other secondary outcomes, including patient-reported assessments.
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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Burst CME: Optimizing Therapy in Parkinson’s Disease
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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 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 & 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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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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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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Clinical ShowCase: Developing a Personalized Treatment Plan for a Patient with Huntington’s Disease Associated Chorea
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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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Overview of New CDC Developmental Milestones for Young Children: Paul Lipkin, MD
August 30th 2022The director of medical outpatient services at the Kennedy Krieger Institute provided insight on the recently announced revisions to the “Learn the Signs. Act Early.” CDC developmental milestones for children. [WATCH TIME: 4 minutes]
Overcoming the Complexities With Gene Therapy in ALS: Anna Underhill, BS
August 18th 2022The postdoctoral researcher at King’s College London provided background on why gene therapies have not seen as much success in treating ALS, and the need for additional validation of genetic mutations. [WATCH TIME: 3 minutes]
BLA Application to Be Submitted for ALS Agent NurOwn Following Phase 3 Statistical Corrections
August 18th 2022The correction in Muscle & Nerve resulted in a statistically significant treatment difference of more than 2 points for average change from baseline in ALSFRS-R in the prespecified efficacy subgroup of with a baseline score of at least 35.
Differences in the Genetic Mutations of ALS: Anna Underhill, BS
August 17th 2022The postdoctoral researcher at King’s College London provided insight on the immunologic differences between genes linked with ALS, and the important aspects to consider for gene-targeted therapies. [WATCH TIME: 3 minutes]
Alnylam Reports Positive Data on Patisiran in APOLLO-B Trial of ATTR Amyloidosis With Cardiomyopathy
August 4th 2022The Alnylam Pharmaceuticals agent, already approved for the treatment of polyneuropathy in those with hATTR amyloidosis, has now shown promise as a therapeutic option for patients with ATTR amyloidosis with cardiomyopathy.
Complexities With SMN2 Copies in Spinal Muscular Atrophy: Basil Darras, MD
August 1st 2022The associate neurologist-in-chief at Boston Children’s Hospital discussed the 4-copy conundrum and whether treatment decisions differ based on SMN2 copies for patients with spinal muscular atrophy. [WATCH TIME: 2 minutes]