FDA news in pediatrics: January 2025
A recap of the FDA submissions and regulatory decisions in pediatrics from January 2025.
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.
FDA accepts sNDA for higher regimen nusinersen for spinal muscular atrophy
The higher dose regimen of nusinersen comprises a more rapid loading regimen, two 50 mg doses 14 days apart, and higher maintenance regimen, 28 mg, every 4 months.
Study: No association between montelukast and neuropsychiatric adverse event risk
"In aggregation with other robust observational studies, these results can inform the management of asthma and allergic rhinitis in this patient group," wrote the study authors.
Using a smartphone application to screen for neonatal jaundice
A good correlation and statistical agreement with total serum bilirubin was observed in a study of a new smartphone-based machine learning app, leaving it a potential neonatal jaundice screening tool.
Survey: titrating trofinetide improves tolerability among Rett syndrome patients
Respondents reported decreased diarrhea severity, improved patient quality of life, and decreased treatment discontinuation among patients with Rett syndrome treated with trofinetide.
