PODCAST: Auditory brain stem implants in young children.

H. Westley Phillips, MD, joined us to provide clinical pearls regarding the early identification of drug-resistant epilepsy patients in the primary care setting.

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.

Parents had a variety of preferences and motivations for acquiring prognostic genetic information about their child, but 4 major findings emerged from parental interviews. Click to read more.