Breakthrough status to Praxis' elsunersen (PRAX-222) for encephalopathy seizure

 

FDA grants Breakthrough Therapy Designation to Praxis' elsunersen (PRAX-222) for SCN2A developmental and epileptic encephalopathy seizures

• Elsunersen (PRAX-222) is an antisense oligonucleotide in development for seizures in SCN2A developmental epileptic encephalopathy.
• The targeted SCN2A developmental and epileptic encephalopathy is specifically caused by gain-of-function variants in the gene.

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