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

ConSynance Therapeutics' CSTI-500, an innovative Triple Monoamine Reuptake Inhibitor, has received FDA's Rare Pediatric Disease Designation for treating Prader-Willi Syndrome in children.

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.

The decision makes pitolisant the first and only FDA-approved, non-scheduled treatment option for daytime sleepiness in this patient population.

The company also announced that it has surpassed its interim analysis enrollment target and expects topline phase 3 data from the FORTIFY study (NCT05775848) among individuals with LGMD21/R9 in 2025.

Approximately half of study participants had tried lifestyle modifications and used vitamin supplementation, and most were taking multiple medications.

The analysis was based on 573 hospitalizations (most patients had an average of 2 hospitalization episodes) in which new BCs were drawn more than 48 hours after initial cultures were negative.