• Praxis Precision Medicines won FDA Breakthrough Therapy designation for elsunersen to treat seizures tied to SCN2A gain-of-function developmental epileptic encephalopathy.
• The designation follows results already presented from a small randomized, sham-controlled early-stage study showing a clinically meaningful drop in seizure burden.
• Benefits appeared durable in an extension phase, supporting a potential disease-targeted approach in a setting with limited treatment options.
• A pivotal EMBRAVE3 registrational study is enrolling on a streamlined single-arm design, with patients treated for 24 weeks followed by a 24-week extension.

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