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Ionis因Angelman综合症药物获得FDA突破性治疗地位

2025-09-09 23:18

Ionis Pharmaceuticals (NASDAQ:IONS) said on Tuesday that the U.S. FDA has granted breakthrough therapy status to its antisense medicine, ION582, for the treatment of angelman syndrome, a neurological disease.
This designation is based on results from a trial of ION582 that showed consistent improvement on domains including communication, cognition and motor function, as well as favorable safety and tolerability.
The breakthrough therapy designation from the FDA is intended to expedite the review of medicines that treat a serious or life-threatening condition.
The stock is up about 3% today.

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