BioMarin· @BioMarin · X·· 4 小时前AI 评分42
AI 导读
今天我们在 @WorldMuscleSoc 年度大会上公布了晚发型庞贝病成人患者的新五年 III 期数据。 这些发现展示了该罕见遗传病患者长期获益，包括既往接受过酶替代治疗和未接受过酶替代治疗的患者。 了解更多所展示数据的信息：http://ms.spr.ly/6012alo1e #WMS2026
正文
Today we announced new five-year Phase 3 data in adults with late-onset Pompe disease at the Annual Congress of the @WorldMuscleSoc.
These findings showcase long-term benefits for individuals living with this rare genetic condition, including people who had previously received enzyme replacement therapy and those who had not.
Learn more about the data presented here: http://ms.spr.ly/6012alo1e
#WMS2026
来源：BioMarin · x.com