Severe brain injury: Helping patient and family on the long road back

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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 this expanded FDA clearance, the wearable becomes the first FDA-cleared, non-drug therapy to treat acute migraines in children.

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.