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Praxis Precision’s Elsunersen Received EMA’s PRIME Designation for SCN2A Developmental and Epileptic Encephalopathy (SCN2A-DEE)

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Praxis Precision’s Elsunersen Received EMA’s PRIME Designation for SCN2A Developmental and Epileptic Encephalopathy (SCN2A-DEE)

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  • The PRIME designation was based on part 1 of the EMBRAVE study evaluating the safety & tolerability of elsunersen in a cohort study on pediatric patients aged 2 to 18yrs. for 13wks.
  • The result of the study demonstrated reduction in seizures & improvement in seizure-free days along with preclinical data. The drug has already received ODD from the FDA & EMA, additionally RPD from the FDA for SCN2A-DEE treatment
  • Elsunersen is a type of antisense oligonucleotide (ASO) developed to specifically decrease the expression of the SCN2A gene. The program is currently being carried out in collaboration with Ionis Pharmaceuticals and RogCon

Ref: BioSpace | Image: Praxis Precision

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Shivani Chandra

Shivani was a content writer at PharmaShots. She has a keen interest in recent innovations in the life sciences industry. She was covering news related to Product approvals, clinical trial results, and updates. We can be contacted at connect@pharmashots.com.

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