Nature Medicine, Published online: 14 April 2026; doi:10.1038/s41591-026-04314-9 Two individuals with KCNT1-related epileptic encephalopathy were treated with a KCNT1-targeting antisense oligonucleotide, leading to significant reductions in seizure frequency and intensity, but also the development of hydrocephalus.
Nature Medicine published a clinical update in Research Highlights on 14 Apr 2026. The item focuses on Antisense oligonucleotide-mediated knockdown therapy in two infants with severe KCNT1 epileptic encephalopathy. Open the detail page to review the full original feed content.