St. Jude Children’s Research Hospital reported preclinical evidence for an antisense oligonucleotide (ASO) strategy designed to reverse a neurodevelopmental disorder linked to HNRNPH2 mutations. In Science Translational Medicine, the team described an ASO therapeutic concept aimed at correcting disease-driving biology associated with the ultrarare X-linked condition. The approach is positioned to address a population with developmental delays, seizures, and profound cognitive and motor impairments where current options are limited. While still preclinical, the study provides a mechanistic basis for therapeutic intervention. ASO programs are increasingly used to modulate expression or splicing targets in neurologic disease, and this update reinforces St. Jude’s continued focus on translational pipeline development for pediatric genetic disorders. The work also highlights the growing use of nucleic acid therapeutics to translate genetic diagnosis directly into targeted intervention strategies.