Investing.com -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE)股价盘前上涨2.2%,此前该公司宣布其用于治疗安格尔曼综合征的实验性药物获得美国食品药品监督管理局(FDA)的突破性疗法认定。 FDA基于一项涉及74名4-17岁患者的I/II期研究的初步临床证据,授予GTX-102(apazunersen)特殊地位,这些患者均有完全母源性...
Source LinkInvesting.com -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE)股价盘前上涨2.2%,此前该公司宣布其用于治疗安格尔曼综合征的实验性药物获得美国食品药品监督管理局(FDA)的突破性疗法认定。 FDA基于一项涉及74名4-17岁患者的I/II期研究的初步临床证据,授予GTX-102(apazunersen)特殊地位,这些患者均有完全母源性...
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