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.

Leonard Dragone, MD, PhD, Disease Area Leader at Johnson & Johnson, discusses how nipocalimab is being incorporated into generalized MG care, what long-term data show, and where J&J sees the therapy’s role evolving.

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.