March 20th 2025
A new analysis reported that givinostat, an oral histone deacetylase inhibitor recently approved for DMD, slowed disease progression by approximately 2 years compared with standard care.
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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 and Commentaries™: Visualizing FcRn as a Therapeutic Target in Neurological Disease
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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 the Implications of FcRN-Targeted Therapies on Generalized Myasthenia Gravis
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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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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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Early Trial Data Highlights Potential of Gene Therapy GNT0004 for Duchenne Muscular Dystrophy
March 18th 2025Long-term follow-up data from an early-phase study of an AAV8-based gene therapy for DMD suggest the treatment was well-tolerated and provided sustained biochemical and functional benefits.
Eteplirsen Treatment Significantly Slows Heart Function Decline in Duchenne Muscular Dystrophy
March 17th 2025A recent study shows that eteplirsen, a drug promoting dystrophin production, significantly slowed the decline in heart function, specifically left ventricular ejection fraction, in patients with Duchenne muscular dystrophy.
New Study to Test Subcutaneous Efgartigimod PH20 in Juvenile Myasthenia Gravis
Spanning 14 weeks, the study evaluates age-appropriate dosing, safety, pharmacokinetics, and clinical effects through once-weekly injections of efgartigimod PH20 SC in juvenile myasthenia gravis.
World Sleep Day with Dr. Eric Olson: Advancing Sleep Health and Awareness
On World Sleep Day, the president of the American Academy of Sleep Medicine discussed the importance of sleep for health, the risks of sleep deprivation, nonpharmacologic strategies to improve sleep, and the need for greater awareness of sleep disorders.
Rethinking Rest on World Sleep Day: Neurologist’s Take on Sleep Priorities
Anne Marie Morse, DO, director of Child Neurology and Pediatric Sleep Medicine at Geisinger Janet Weis Children’s Hospital, dove into the importance of sleep health, its impact on brain function, and emerging research in honor of World Sleep Day.
Misdiagnosed Migraine Leads to Significant Burden on Healthcare Resource Utilization
New research highlights the significant economic burden of misdiagnosed migraine patients, revealing higher healthcare costs and resource utilization over time compared to those with accurate diagnoses.