A UC San Diego-led team reported in Nature Medicine that personalized antisense oligonucleotides reduced seizure burden in two children with SCN2A-related developmental and epileptic encephalopathies, with one patient able to walk independently. The investigators designed individualized ASOs for a nine-year-old boy with a gain-of-function SCN2A variant and a 14-year-old with a mixed loss- and gain-of-function variant after seizures failed to respond to more than 10 antiseizure medications.
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