8 questions for a thorough history to diagnose migraines

The submission to the FDA includes data from the phase 2b ReNeu trial of patients aged 2 years and older with NF1-associated PN causing significant morbidity.

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.