Epilepsy

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.

Here's some of what is coming soon to NeurologyLive® this week.

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.

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At a NeurologyLive Clinical Forum in Philadelphia, Jessica D. Schulte, MD, PhD, of NYU Grossman School of Medicine, discussed how earlier recognition and systemic therapy options are changing the treatment paradigm for patients with NF1-associated plexiform neurofibromas.

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EpilepsyGTx and Viralgen have entered a manufacturing collaboration to support development of EPY201, an investigational AAV9-based gene therapy for focal refractory epilepsy planned to enter first-in-human testing in 2027.

Here's some of what is coming soon to NeurologyLive® this week.

The FDA extended its review of relutrigine for SCN2A- and SCN8A-related epilepsies by three months, pushing the PDUFA date to December 27, 2026.

The FDA lifted a clinical hold on simufilam, clearing Filana Therapeutics, formerly Cassava Sciences, to begin a phase 2a study in TSC-related epilepsy.

Xenon Pharmaceuticals submitted an FDA new drug application for azetukalner, a KV7 potassium channel opener, as adjunctive therapy for focal seizures in epilepsy.

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Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending September 18, 2026.

Marketed as Fayuvi, the 1-time AAV9 gene therapy showed sustained cerebrospinal fluid heparan sulfate drops and developmental gains among pediatric patients with mucopolysaccharidosis type IIIA in prior studies.

Amy Waldman, MD, Medical Director of the Leukodystrophy Center at Children's Hospital of Philadelphia, discusses zilganersen's mechanism, safety profile, clinical considerations, and what the first-ever approval for Alexander disease means for the broader leukodystrophy field.

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Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending September 11, 2026.

An indirect comparison found zorevunersen-treated patients with Dravet syndrome showed greater gains in adaptive functioning and behavior than natural history patients.

Here's some of what is coming soon to NeurologyLive® this week.

FDA approved Zanvastro (zilganersen), the first therapy to directly target the GFAP protein buildup that drives Alexander disease, based on a phase 3 trial showing improved walking speed and motor function versus untreated controls.

The phase 3 Aspire study of apazunersen did not meet its primary cognition end point or key secondary end point in Angelman syndrome.

Test your neurology knowledge with NeurologyLive®'s weekly quiz series, featuring questions on a variety of clinical and historical neurology topics. This week's topic is pediatric epilepsy care!

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending August 28, 2026.