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Rare Disease Therapies Advance for Dravet Syndrome and Spinal Muscular Atrophy

Mar 05, 2026

New treatments for rare pediatric neurological conditions show promise, with zorevunersen reducing seizures in Dravet syndrome patients and an SMA therapy under FDA review following stem cell research breakthroughs.

Stoke Therapeutics Reports Durable Dravet Data, Expects Phase 3 Enrollment Completion in Q2 2026

Feb 23, 2026

Stoke Therapeutics presented long-term Dravet syndrome data showing up to 80% seizure reductions sustained through three years, outlined its Phase 3 EMPEROR trial design targeting mid-2027 data, and disclosed first patient dosing in its ADOA program.