Primary care physicians, including pediatricians, admit that they are not as familiar about specific aspects of epilepsy as they should be.
Michael Kruer, MD explains genetic testing and cerebral palsy
Genetic research is revealing that up to 35% of cerebral palsy cases have an identifiable genetic cause, leading to earlier diagnoses and potential precision medicine treatments.
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.
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.
Apitegromab BLA submitted to FDA for spinal muscular atrophy
Application submission is based on positive phase 3 data that demonstrated improvement in patients with type 2 and 3 SMA receiving current standard of care.
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.