Between the bluntness of gene editing and the palliation of symptom management, medicine has long sought a middle path — one that addresses the source of suffering without rewriting the book of life itself. In a recent study, two boys with severe SCN2A-related epilepsy received personalized antisense therapy designed to silence only their broken gene copies, leaving healthy ones intact. One eliminated his seizures entirely and began to walk unassisted; the other discontinued medication and regained language and motor skills that had long been out of reach. The case raises a quiet but urgent qu
Allele-Specific Antisense Therapy Shows Promise in SCN2A Epilepsy Patients
Two children with severe epileptic encephalopathy experienced daily seizures and developmental delays before treatment, with one achieving complete seizure elimination and improved motor and language skills.