
Trofinetide shows significant real-world improvements in Rett syndrome symptoms for pediatric and adult patients, enhancing communication and quality of life.
Trofinetide shows significant real-world improvements in Rett syndrome symptoms for pediatric and adult patients, enhancing communication and quality of life.
The pediatrician-in-chief at Johns Hopkins Children’s Center shared her reaction to receiving this year’s Bernard Sachs Award in recognition of her contributions to child neurology. [WATCH TIME: 5 minutes]
A case series reveals safe use of vamorolone and prednisolone during microdystrophin gene therapy for Duchenne muscular dystrophy in two boys.
A recent study reveals the significant economic burden of Angelman syndrome, highlighting rising healthcare costs and ongoing therapeutic developments.
At CNS 2025, the executive director at the Child Neurology Foundation presented on the importance of collaboration among clinicians and advocacy groups to connect families with care support services. [WATCH TIME: 4 minutes]
A post hoc analysis of the phase 3 EPIDYS trial presented at CNS 2025 revealed that givinostat delayed disease progression in 2 distinct age groups of patients with Duchenne muscular dystrophy.
A recent analysis highlights the link between vastus lateralis fat fraction and treatment outcomes in Duchenne muscular dystrophy patients using givinostat.
Joseph Vithayathil, MD, discusses being awarded the Elterman Research Grant at the 2025 CNS Annual Meeting in Charlotte, North Carolina.
The BeeLine trial is a placebo-controlled study designed to evaluate radiprodil’s impact on both seizure frequency and non-seizure symptoms associated with GRIN-NDD.
Analysis of long-term safety of intranasal diazepam shows favorable results in children and adolescents with cluster or acute, repetitive seizures.
Study results show that deflazacort tops prednisone across multiple checkpoints in patients with Duchenne muscular dystrophy.
Epilepsy-specific TRAQ questionnaire shows high validity in pediatric epilepsy patients transitioning to adult epilepsy care.
Data presented at the Child Neurology Society Annual Meeting suggest that the safety and efficacy of cannabidiol (Epidiolex; GW Pharmaceuticals) is maintained through 72 weeks of treatment for patients with LGS.
The drug recently gained FDA approval for treatment of lower limb spasticity in pediatric patients without cerebral palsy, in which it is associated with improved functional outcomes.
A pooled analysis of 2 phase 3 trials of fenfluramine demonstrate the clinical potential of the drug for patients with Dravet syndrome.
The professor and head of the Department of Neurology and Rehabilitation at the University of Illinois at Chicago spoke about how data can better inform epilepsy care.
The Carolyn and Matthew Bucksbaum Professor of Clinical Ethics at the University of Chicago spoke about the ethical considerations of spinal muscular atrophy and its treatment.
The clinical professor in pediatric neurology and director of regional neurology services at Children's Hospital Colorado shared her thoughts on why epilepsy is the perfect paradigm to implement telemedicine.
The Pediatric Movement Disorders Neurologist at UT Southwestern recommends that when encountering a patient with a movement disorder to organize your thought process in 3 steps.
Darryl De Vivo, MD, emphasized that while there's been a tremendous advance in the field of SMA, it’s important to continue tinkering around the margins to make the effective treatment increasingly better.
The Sidney Carter Professor of Neurology at Columbia University Medical Center spoke about the 127 year history of spinal muscular atrophy and how basic science has led to exciting developments.