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.
July 25th 2024
Over a 48-week period, patients treated with low molecular weight dextran sulfate compound had minimal changes in ALS Functional Rating Scale and ALS Assessment Questionnaire.
Evolving Perspectives in Alzheimer's Disease: Reaching an Earlier Diagnosis, Understanding Neuroinflammation, ...
September 16, 2024
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Cases and Conversations™: Applying Best Practices to Prevent Shingles in Your Practice
October 16, 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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Community Practice Connections™: Transforming Multiple Sclerosis Care – Clinical Updates on the Effects of BTK Inhibitors
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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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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 Proteins in Neurologic Disorders
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Burst Expert Illustrations & Commentary™: Visualizing the Role of the Complement Pathway in Neurological 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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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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Previewing ALS Association’s Inaugural Nexus Conference: John Novak, MD, MS
June 28th 2024The director of the OhioHealth ALS Clinic and vice chair of the ALS Association Care Services Committee discussed the upcoming ALS Nexus Conference and how themes of the event align with the direction of clinical care. [WATCH TIME: 3 minutes]
Unseen Battles: Advocacy and Community in the Face of Myasthenia Gravis
June 28th 2024Anaya, a young woman with myasthenia gravis, discusses her challenges and triumphs living with a rare disease. She shares her personal story of advocacy and community, finding strength in the disease's impact on her independence and identity.
Pitolisant Reduces Excessive Daytime Sleepiness and Fatigue in Myotonic Dystrophy Type 1
June 27th 2024Additional data of a phase 2 study presented at the 2024 SLEEP Annual Meeting demonstrated that pitolisant significantly improved in secondary end points among patients with myotonic dystrophy type 1.
Managing Clinical Stability in Adults Spinal Muscular Atrophy Patients
Clinical experts in spinal muscular atrophy (SMA) address the issue of patient disengagement from healthcare services, which can occur due to a perceived lack of disease progression or stabilization, leading to undetected disease advancement and increased risk to patients, and provide recommendations for encouraging patients to re-engage with medical care.
Key Players in Transitioning Patients with SMA from Pediatric Care to Adult Health Care
Key opinion leaders (KOLs) further explore the transition of care from pediatric to adult healthcare systems for patients with spinal muscular atrophy (SMA), emphasizing the essential role of multidisciplinary support in ensuring a successful transition.
New HEALEY-ALS Trial Results Show CNM-Au8’s Effect on Survival, Neurofilament Light
June 19th 2024CNM-Au8, an oral suspension of clean-surfaced, catalytically-active gold nanocrystals, resulted in prolonged survival and reductions in neurofilament light, a prominent biomarker of neuroaxonal damage.
NeuroVoices: Alessio Travaglia, PhD, on New FNIH Partnership to Accelerate ALS Biomarkers, Research
June 19th 2024The director at the Foundation for the National Institutes of Health provided commentary on a recently launched partnership aimed at building a repository of ALS data to advance drug development for the disease.
Phase 1/2 Studies Highlight Durable Impacts of Gene Therapy TSHA-102 in Rett Syndrome
June 18th 2024Initial data from a few pediatric and adult patients with Rett syndrome showed that treatment with TSHA-102 resulted in numerous positive enhancements across multiple efficacy measures and clinical domains.
Key opinion leaders (KOLs) address the challenges and obstacles associated with transitioning patients from pediatric to adult care as they age out of the pediatric system, offering valuable insights and strategies for successfully navigating this process.
Presentation of Adult Spinal Muscular Atrophy in Clinics
Leading medical professionals specializing in spinal muscular atrophy (SMA) discuss the common clinical presentations of undiagnosed or newly diagnosed adult patients, highlighting crucial signs and symptoms that healthcare providers should recognize.
Reaction and Benefits of Deutetrabenazine One Pill, Once-Daily Tablet Approval
June 15th 2024Rakesh Jain, PhD, MS, clinical professor of psychiatry at the Texas Tech University School of Medicine, provided insight on a recently approved tablet dosage for deutetrabenazine, an FDA-approved therapy for chorea and tardive dyskinesia associated with Huntington disease.
Newly Formed Clinical Advisory Board United in Potential of SAT-3247 for Duchenne Muscular Dystrophy
Published: June 13th 2024 | Updated: June 14th 2024Satellos Bioscience has established a clinical advisory board to aid in developing their lead drug candidate SAT-3247, an oral small molecule therapy for Duchenne muscular dystrophy.
Mirdametinib Produces Sustained Reduction of Plexiform Neurofibroma in Phase 2b ReNeu Trial
June 13th 2024For up to 24 months, patients on mirdametinib demonstrated significant improvements in pain severity, pain interference, and health-related quality of life, in addition to deep and durable plexiform neurofibroma volume reductions.
Gene Therapy GNT0004 Demonstrates Early Efficacy, Safety in Duchenne Muscular Dystrophy
June 12th 2024Although a small sample of 5 patients with DMD, results showed significant expression of microdystrophin and reductions in creatine kinase, a biomarker of muscular distress, in GTN0004-treated patients.