2756001-75-7
2756001-75-7
Chemical Structure
Elsunersen
Synonym(s): PRAX-222
- CAS No.: 2756001-75-7
- Formula:C230H320N67O126P19S13
- Molecular Weight:7044.71
SMILES: [Elsunersen]
Biological Activity: Elsunersen (PRAX‑222) is a gapmer antisense oligonucleotide targeting SCN2A mRNA. Elsunersen reduces the protein level of the voltage-gated sodium channel NaV1.2 by degrading SCN2A mRNA, alleviating neuronal network hyperexcitability. Elsunersen combined with sodium channel blockers terminates refractory status epilepticus and reduces seizure frequency. Elsunersen can be used in research related to epileptic encephalopathy[1][2].
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Elsunersen | Elsunersen (PRAX‑222) is a gapmer antisense oligonucleotide targeting SCN2A mRNA. Elsunersen reduces the protein level of the voltage-gated sodium channel NaV1.2 by degrading SCN2A mRNA, alleviating neuronal network hyperexcitability. Elsunersen combined with sodium channel blockers terminates refractory status epilepticus and reduces seizure frequency. Elsunersen can be used in research related to epileptic encephalopathy. | |||||||||||||||||||||
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References
- [1]. Frizzo S, et al. Complementary Antisense Oligonucleotide Treatment and Precision Sodium Channel Modulation for Early Onset SCN2A Developmental and Epileptic Encephalopathy: Emergency Use Case in a Preterm Infant with Refractory Status Epilepticus (P10-11.009). Neurology. 2026;106(11 Suppl 1):2271.
- [2]. Potschka H, et al. Drug discovery and preclinical testing of drug candidates for developmental and epileptic encephalopathies. Epilepsia. 2025 Dec;66(12):4597-4623.
