Relutrigine is an investigational medicine that can be taken orally or administered through a G/J tube. Relutrigine is designed to regulate sodium flow in brain cells by targeting overactive sodium channels that cause seizures; therefore, potentially offering better seizure control with fewer side effects. Relutrigine has been designed to maximize its effects against overactive sodium channels that are believed to cause seizure activity while minimizing the blocking of normal activity needed for healthy brain function.

Elsunersen is an investigational antisense oligonucleotide (ASO) administered monthly by intrathecal injection. Elsunersen has the potential to be the first disease-modifying treatment for early-onset gain-of-function SCN2A DEE, designed to selectively decrease SCN2A gene expression. By targeting the underlying genetic cause of disease, elsunersen has demonstrated potential to go beyond seizures to treat other symptoms of the disease.