Scn8a Antisense Oligonucleotide Is … – Wiley Online Library

Objective. SCN8A encephalopathy is a developmental and epileptic encephalopathy (DEE) caused by de novo gain‐of‐function mutations of sodium channel Na v 1.6 that result in neuronal hyperactivity. Affected individuals exhibit early onset drug‐resistant seizures, developmental delay, and cognitive impairment. This study was carried out to determine whether reducing the abundance of the …

https://onlinelibrary.wiley.com/doi/abs/10.1002/ana.25676