Praxis Precision Medicines, Inc. 宣布，其研发的药物Elsunersen已获得美国食品药品监督管理局（FDA）授予的突破性疗法认定。该认定针对的是由SCN2A基因功能获得性变异所引起的发育性和癫痫性脑病（DEE）相关的癫痫发作治疗。
突破性疗法认定旨在加速用于治疗严重或危及生命疾病的新药开发和审评。对于Praxis而言，这一认定标志着其针对这一罕见且严重神经系统疾病的创新疗法开发取得了重要进展。
Praxis Precision Medicines, Inc. 宣布，其研发的药物Elsunersen已获得美国食品药品监督管理局（FDA）授予的突破性疗法认定。该认定针对的是由SCN2A基因功能获得性变异所引起的发育性和癫痫性脑病（DEE）相关的癫痫发作治疗。
突破性疗法认定旨在加速用于治疗严重或危及生命疾病的新药开发和审评。对于Praxis而言，这一认定标志着其针对这一罕见且严重神经系统疾病的创新疗法开发取得了重要进展。
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