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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Mechanistic Potential of Taldefgrobep Alfa in Spinal Muscular Atrophy: Lindsey Lee Lair, MD
July 28th 2022The vice president of clinical development at Biohaven shed light on a newly initiated phase 3 study evaluating a promising agent for patients with all types of spinal muscular atrophy. [WATCH TIME: 2 minutes]
Assessing Treatment Options and Improving Utilization for Spinal Muscular Atrophy: Basil Darras, MD
July 28th 2022The associate neurologist-in-chief at Boston Children’s Hospital provided insight on the treatment decisions clinicians make for patients with SMA and the challenges with improving optimization. [WATCH TIME: 3 minutes]
Prescribing Psychical Activity Regimens in Spinal Muscular Atrophy: Jacqueline Montes, PT, EdD
July 26th 2022The associate professor of rehabilitation and regenerative medicine at Columbia University Medical Center discussed the ways clinicians go about properly prescribing exercise regimens for patients with spinal muscular atrophy. [WATCH TIME: 3 minutes]
European Commission Passes Eladocagene Exuparvovec as First Approved Treatment for AADC Deficiency
July 22nd 2022In previous clinical studies, patients with AADC deficiency treated with the recombinant AAV2-based gene therapy achieved motor milestones, including head control and unassisted sitting.
Treating Familial ALS at the Source: The Introduction of Gene Therapies
July 20th 2022As scientific researchers continue to uncover new genetic links to amyotrophic lateral sclerosis, the rise of gene therapies to treat the neuromuscular disease will remain a development to watch in the coming years.
NeuroVoices: Lindsey Lee Lair, MD, on the Phase 3 RESILIENT Trial of Taldefgropeb Alfa in SMA
July 20th 2022The vice president of clinical development at Biohaven provided insight on a new phase 3 study assessing a muscle-targeted agent as an adjunct therapy to currently approved spinal muscular atrophy medications.
Muscle-Directed Therapy Apitegromab Reports Positive 24-Month Results in Spinal Muscular Atrophy
July 12th 2022Over a 2-year period, apitegromab-treated patients demonstrated sizable and sustained improvements in Hammersmith Functional Motor Scale-Expanded and substantial increases in Revised Upper Limb Module scores.
Biohaven Commences Phase 3 Study of Taldefgrobep Alfa in Spinal Muscular Atrophy
July 11th 2022Taldefgrobep alfa, a muscle-targeted experimental treatment potentially used in combination with other SMA therapies, will be evaluated in a cohort of 180 patients with SMA, regardless of ambulatory status or disease classification.
NeurologyLive® Top Expert Interviews: June 2022
July 7th 2022Expert clinicians offer their insight on the sleep disorder landscape, the state of mental health in pediatric migraine, an inaugural movement disorders congress, a novel stroke rehabilitation tool, and psychedelics in cluster headache.