October 8th 2025
The BeeLine trial is a placebo-controlled study designed to evaluate radiprodil’s impact on both seizure frequency and non-seizure symptoms associated with GRIN-NDD.
New research finds significant challenges for children with disabilities in foster care
September 27th 2024A study presented at the American Academy of Pediatrics 2024 National Conference finds children with disabilities in foster care face lower permanency rates and higher mortality risks, calling for specialized support and interventions.
Cerliponase alfa FDA approved to slow loss of ambulation in children of all ages with CLN2 disease
July 25th 2024From baseline to final assessment, all 7 matched cerliponase alfa-treated children under 3 years of age maintained a motor score of 3, representing a "grossly normal gait, signifying a delay in disease onset," stated BioMarin.
Paternal depression associated with adverse childhood experiences in children
October 21st 2023In this Q&A, Kristine H. Schmitz, MD, explains her team's recent research on paternal depression and adverse childhood experiences during the 2023 American Academy of Pediatrics National Conference & Exhibition.
Exome sequencing should be used for diagnosing cerebral palsy
October 13th 2023A systematic review and meta-analysis found that such sequencing indeed has an equivalent high genetic diagnostic yield in cerebral palsy, which supports including this neurodevelopmental disorder among those for which diagnostic exome sequencing should be used.