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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Importance of National Women Physicians Day and Valuing Women in Neurology: Jan Brandes, MD, MS
February 3rd 2022The assistant clinical professor at Vanderbilt University School of Medicine shared what National Women Physicians Day means to her, and the history behind its celebration. [WATCH TIME: 6 minutes]
Alternative Career Paths: Clinical Care Through Clinical Research
February 3rd 2022For women, the decision to opt for an unconventional career path can be inspired by conflicting responsibilities and balancing life with work. But, as Sana Syed, MD, MPH, explains, following your passion is still an achievable dream despite these challenges.
Blarcamesine Shows Promise as Potential Rett Syndrome Treatment, New Data Indicate
February 2nd 2022Treatment with blarcamesine resulted in statistically significant improvements on the new primary end point and significantly reduced Rett syndrome symptoms through changes in potential biomarkers of disease pathology.
Risdiplam Gets Priority Review for Presymptomatic SMA in Babies Under 2 Months
January 25th 2022The submission to the FDA is supported by interim data from the RAINBOWFISH trial (NCT03779334), as all 5 babies treated with risdiplam maintained the ability to swallow and feed exclusively orally after 12 months.
Compassionate Use Clinical Trial of FGF-1 Initiated for Amyotrophic Lateral Sclerosis
January 19th 2022Zhittya Genesis Medicine announced the trial to evaluate its biological drug, FGF-1, which is administered via an intranasal delivery device, in a cohort of individuals with mild to moderate ALS.
Long-Term IV Edaravone Not Associated With Disease-Modifying Benefit in ALS
January 13th 2022Despite not providing additional, clinically relevant benefit for patients with amyotrophic lateral sclerosis as an add-on to standard therapy, the intravenous treatment was feasible and well tolerated.
Spasticity, Dystonia Commonly Under-Identified in Children With High Cerebral Palsy Risk
January 13th 2022Only 30% of the observed cohort of more than 150 children had their hypertonia type specified as spasticity and/or dystonia by age 5 years, highlighting a need for additional education efforts.