Tailoring prenatal iron supplementation according to the mother’s haemoglobin levels and baseline iron stores leads to improved cognitive functioning in children at 4 years of age, according to a study.
Revumenib, a novel potent, selective oral inhibitor of the menin-KMT2A interaction, demonstrated promising antileukaemic activity in children and adults with highly refractory acute leukaemia with KMT2Ar* or NPM1* mutation, a phase I dose-escalation study finds.
A single dose of nirsevimab administered before or during the respiratory syncytial virus (RSV) season provides protection against RSV lower respiratory tract infection (LRTI) hospitalization and very severe infection in infants, according to the preliminary results of a clinical trial conducted in real-world settings.
Children with Lennox-Gastaut syndrome (LGS) and Dravet's syndrome (DS) achieve improvements in both seizure management and nonseizure outcomes following treatment with cannabidiol (CBD)—a success echoed in their adult counterparts, as shown in a study presented at AAN 2023.
Treatment with ataluren improves ambulatory function, as assessed by the 6-minute walk distance (6MWD) test, among boys with nonsense mutation Duchenne muscular dystrophy (nmDMD), according to the subgroup analysis of Study 041 trial presented at AAN 2023.