Impact of rapid treatment for pediatric status epilepticus (VIDEO)

H. Westley Phillips, MD, explains why awareness and availability of epilepsy surgery is important for pediatric providers among drug-resistant epilepsy patients.

Jamie Glater, MD, discusses an NIH-sponsored study investigating the effectiveness of an auditory brain stem implant in young children with congenital cochlear nerve agenesis.

With the decision, Kebilidi becomes the first FDA-approved gene therapy for AADC deficiency, and is indicated for adult and pediatric patients.

Data stems from the first 4 patients dosed in a low-dose cohort of an ongoing phase 1/2 open-label trial.

The TrustTSC trial did not achieve its primary endpoint of percent reduction in 28-day frequency of TSC-associated seizures.

The approved indication is for patients aged 7 years and up with narcolepsy to treat cataplexy or excessive daytime sleepiness.