Stoke Therapeutics 的 zorevunersen 治疗 Dravet 综合征获得 FDA 突破性地位。 Stoke Therapeutics' zorevunersen gets FDA breakthrough status for treating Dravet syndrome.

Stoke Therapeutics 已获得 FDA 的 zorevunersen 突破性疗法认定，zorevunersen 是一种治疗与 SCN1A 基因突变相关的 Dravet 综合征的实验性药物。 Stoke Therapeutics has received FDA's Breakthrough Therapy Designation for zorevunersen, an experimental drug for treating Dravet syndrome linked to the SCN1A gene mutation. 该公司计划讨论一项全球第三阶段研究,并打算到年底更新其计划,针对的是Zorevurnersen,作为Dravet综合症的首种疾病调控药物。 The company plans to discuss a global Phase 3 study and aims to update its plans by year-end, targeting zorevunersen as the first disease-modifying medication for Dravet syndrome. 早期研究显示,发作频率显著降低. Early studies showed significant reductions in seizure frequency.