Child Neurology Society Annual Meeting (CNS)

Newly presented post hoc analyses examined diazepam nasal spray for status epilepticus in seizure clusters and intervals between clusters in children, alongside real-world data on rescue medication prescribing.

A retrospective 10-center U.S. study found stiripentol reduced convulsive seizure frequency, status epilepticus episodes, and emergency health care use in patients with Dravet syndrome.

A phase 1b/2 trial is testing an intrathecal antisense oligonucleotide designed to lower KCNT1 expression in children with a severe, drug-resistant epilepsy syndrome.

Ocrelizumab demonstrated noninferiority to fingolimod for relapse control but displayed greater reductions in MRI lesion activity among pediatric patients with relapsing-remitting multiple sclerosis.

Pivotal AFFINITY DUCHENNE results showed microdystrophin expression above the prespecified threshold in 93% of evaluable participants, with preliminary functional findings favoring treatment over external controls.

Three-year extension data from the phase 3 IB1001-301 trial show levacetylleucine slowed disease progression in both adults and children with Niemann-Pick disease type C.

All 10 REVEAL Part A participants gained or regained at least 1 developmental milestone after a single intrathecal TSHA-102 dose, with no treatment-related SAEs reported.

Findings from the phase 3 METEOROID trial showed that satralizumab reduced the risk of relapse in patients with relapsing myelin oligodendrocyte glycoprotein antibody-associated disease.

The phase 1 ASCEND trial is testing a single intrathecal dose of the investigational AAV9 gene therapy INS1201 in young boys with Duchenne muscular dystrophy.

An analysis of the phase 3 GEMZ trial presented at CNS 2026 showed that fenfluramine produced comparable seizure reductions and safety outcomes in children and adults with CDKL5 deficiency disorder.

A post hoc analysis of the phase 2/3 NPC002 trial presented at CNS 2026 showed that the benefit of arimoclomol plus miglustat in Niemann-Pick disease type C emerged as early as 3 months after treatment initiation.

The Galassia trial will evaluate the safety and tolerability of intranasal cenegermin in about 60 children with spastic cerebral palsy across Italy.

The chief medical officer at Mandos Health discussed new CNS 2025 data on adrabetadex in infantile-onset Niemann-Pick type C and the growing evidence of its potential disease-modifying impact.

Experts shared their clinical perspectives on trending topics in the treatment and management of multiple sclerosis at the 54th Child Neurology Society (CNS) Annual Meeting, held October 8-11, 2025.

At CNS 2025, the associate professor of neurology and pediatrics at the University of Virginia discussed using educational model frameworks to prepare child neurology trainees for clinical practice. [WATCH TIME: 3 minutes]

James N. Brenton, MD, director of the Pediatric MS and Related Disorders Clinic at the University of Virginia, discussed how lifestyle modification and multidisciplinary care can support disease management in children with multiple sclerosis.

At CNS 2025, the program director of child neurology residency at Boston Children's Hospital discussed strategies to expose medical students to child neurology early in their clinical training. [WATCH TIME: 4 minutes]

New findings reveal early treatment with Trappsol Cyclo shows promise for young patients with Niemann-Pick disease type C1, enhancing safety and efficacy.

A recent review of studies over the past decade, presented at CNS 2025, suggests that the ketogenic diet can significantly reduce seizure frequency in children with drug-resistant epilepsy.

A new study reported that children and adolescents with Tourette syndrome on dopamine D2 receptor antagonists had higher rates of psychiatric and metabolic adverse events than those unexposed.

The associate professor of neurology and pediatrics at the University of California, San Francisco, discussed updated recommendations for continuous EEG use in neonates, highlighting risk stratification, and the growing importance of family involvement. [WATCH TIME: 3 minutes]

Neurology News Network. for the week ending October 18, 2025. [WATCH TIME: 4 minutes]

Anne O’Donnell-Luria, MD, PhD, a practicing clinical geneticist at Boston Children’s Hospital, sat down at CNS 2025 to discuss advances in genomic diagnostics, variant interpretation, and the expanding impact of neurogenetic discovery on clinical practice.

At the 2025 CNS Annual Meeting, Craig Press, MD, discussed tailoring neuroICU treatment for pediatric patients, advances in seizure management, and how multidisciplinary collaboration can improve outcomes.

Apitegromab, an invesigational muscle-targeting treatment, showed promising results in improving motor function among young patients with SMA in a phase 2 trial.

At CNS 2025, the child neurologist at Rady Children's Hospital-San Diego talked about the expanding role of genetics for diagnosis and treatment in pediatric neurology. [WATCH TIME: 5 minutes]

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.