FDA expands use of eculizumab to patients 6 years and up with generalized myasthenia gravis
First approved for PNH in 2007, the monoclonal antibody is now the first and only treatment for pediatric patients living with gMG.
PODCAST: Auditory brain stem implants in young 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.
Weekly review: Several key FDA approvals, new puzzler case, and more
Get caught up with Contemporary Pediatrics! This list helps you navigate our top stories from the week, all in one place.
FDA approves non-invasive risdiplam tablet for spinal muscular atrophy
The new risdiplam tablet is suitable for those aged 2 years and older who weigh more than 44 lbs, according to Genentech.
Positive data reported for Trappsol Cyclo in patients 3 years and under with NPC1
Cyclo Therapeutics has enrolled 10 patients in a single-arm sub-study treating newborns to 3 years of age, evaluating the Trappsol Cyclo in the youngest subsets.
FDA news in pediatrics: January 2025
A recap of the FDA submissions and regulatory decisions in pediatrics from January 2025.
