2024年FDA批准的50款新药概览 患者 治疗 药物 新药 领域 -健康界
189.00 US$
Published date: January 14, 2026
- Location: Nr27 5zg, Amla, Gifu, United Kingdom
与此前FDA批准的另一款SMA基因疗法Spinraza相比，Zolgensma（AVXS-101）无论在疗效还是成本效益上都具有优势。 Givosiran（Givlaritm）用于治疗成人急性肝卟啉症，这是一种导致有毒卟啉分子积聚的遗传性疾病。 Givosiran是一个双链siRNA，由Alnylam开发的。 2017年，FDA批准Keytruda作为"不限癌种"（也被称为广谱抗癌药）疗法治疗微卫星不稳定性高（MSI-H）或错配修复缺陷（dMMR）实体瘤患者，这是FDA批准的首款不依照肿瘤来源，而是依照生物标志物进行区分的抗肿瘤疗法。 跨性别色情片 • Scenesse（afamelanotide），用于治疗红细胞生成性原卟啉症患者，可提供系统性光保护作用以预防光毒性。 这是一种罕见的情况，由通常有助于红细胞向身体输送氧气的化学物质的过量积累引起；即使短暂的日晒也会导致皮肤疼痛。 •.
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